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Reference Publications
SERVICE
Genome Editing Services for Cell Lines
CRISPR Cell Line Knock-Out, Knock-in, Point Mutation
- Peluso, M. O., Adam, A., Armet, C. M., Zhang, L., O’Connor, R. W., Lee, B. H., … & Palombella, V. J. (2020). The Fully human anti-CD47 antibody SRF231 exerts dual-mechanism antitumor activity via engagement of the activating receptor CD32a. Journal for ImmunoTherapy of Cancer, 8(1), e000413.
- Tanic, J., Wang, Y., Lee, W., Coelho, N. M., Glogauer, M., & McCulloch, C. A. (2019). Adseverin modulates morphology and invasive function of MCF7 cells. Biochimica et Biophysica Acta (BBA)-Molecular Basis of Disease, 1865(10), 2716-2725.
- Panda, D., Gjinaj, E., Bachu, M., Squire, E., Novatt, H., Ozato, K., & Rabin, R. L. (2019). IRF1 maintains optimal constitutive expression of antiviral genes and regulates the early antiviral response. Frontiers in immunology, 10, 1019.
- Pisapia, P., Malapelle, U., Roma, G., Saddar, S., Zheng, Q., Pepe, F., … & Nikiforov, Y. E. (2019). Consistency and reproducibility of next‐generation sequencing in cytopathology: A second worldwide ring trial study on improved cytological molecular reference specimens. Cancer cytopathology, 127(5), 285-296.
- Ilic, D. (2019). Latest developments in the field of stem cell research and regenerative medicine compiled from publicly available information and press releases from nonacademic institutions in October 2018. Regenerative medicine, 14(2), 85-92.
- Simkin, D., Searl, T. J., Piyevsky, B. N., Forrest, M., Williams, L. A., Joshi, V., … & Penzes, P. (2019). Impaired M-current in KCNQ2 Encephalopathy Evokes Dyshomeostatic Modulation of Excitability. bioRxiv, 538371. https://doi.org/10.1101/538371
- Jang, Y., Choi, J., Park, N., Kang, J., Kim, M., Kim, Y., & Ju, J. H. (2019). Development of immunocompatible pluripotent stem cells via CRISPR-based human leukocyte antigen engineering. Experimental & Molecular Medicine, 51(1), 3.
- Colomar-Carando, N., Meseguer, A., Jutz, S., Herrera-Fernández, V., Olvera, A., Kiefer, K., … & Vicente, R. (2018). Zip6 Transporter Is an Essential Component of the Lymphocyte Activation Machinery. The Journal of Immunology, ji1800689.
- Tanic, J. (2018). A Role for Adseverin in the Invasion and Migration of MCF7 Breast Adenocarcinoma Cells (Doctoral dissertation).
- Lizarraga, S. B., Maguire, A. M., Ma, L., van Dyck, L. I., Wu, Q., Nagda, D., … & Cowen, M. H. (2018). Human neurons from Christianson syndrome iPSCs reveal allele-specific responses to rescue strategies. bioRxiv, 444232.
- Tanaka, H., Kondo, K., Chen, X., Homma, H., Tagawa, K., Kerever, A., … & Fujita, K. (2018). The intellectual disability gene PQBP1 rescues Alzheimer’s disease pathology. Molecular Psychiatry, 1.
- Yin, Y., Garcia, M. R., Novak, A. J., Saunders, A. M., Ank, R. S., Nam, A. S., & Fisher, L. W. (2018). Surf4 (Erv29p) binds amino-terminal tripeptide motifs of soluble cargo proteins with different affinities, enabling prioritization of their exit from the endoplasmic reticulum. PLoS biology, 16(8), e2005140.
- Selvan, N., George, S., Serajee, F. J., Shaw, M., Hobson, L., Kalscheuer, V. M., … & Schwartz, C. E. (2018). O-GlcNAc transferase missense mutations linked to X-linked intellectual disability deregulate genes involved in cell fate determination and signaling. Journal of Biological Chemistry, jbc-RA118.
- Smalley, E. (2018). FDA warns public of dangers of DIY gene therapy. https://doi.org/10.1038/nbt0218-119
- Chai, S., Wan, X., Ramirez-Navarro, A., Tesar, P. J., Kaufman, E. S., Ficker, E., … & Deschênes, I. (2018). Physiological genomics identifies genetic modifiers of long QT syndrome type 2 severity. The Journal of clinical investigation, 128(3).
- Boi, S., Ferrell, M. E., Zhao, M., Hasenkrug, K. J., & Evans, L. H. (2018). Mouse APOBEC3 expression in NIH 3T3 cells mediates hypermutation of AKV murine leukemia virus. Virology, 518, 377-384. https://doi.org/10.1016/j.virol.2018.03.014.
- Molinski, S. V., et al. (2017). Orkambi® and amplifier co‐therapy improves function from a rare CFTR mutation in gene‐edited cells and patient tissue. EMBO Molecular Medicine, e201607137.
- Petrovic, P. B. (2017). Myosin Phosphatase Rho-interacting Protein Regulates DDR1-mediated Collagen Tractional Remodeling (Doctoral dissertation, University of Toronto (Canada)).
- Peng, L., Zhang, H., Hao, Y., Xu, F., Yang, J., Zhang, R., … & Chen, C. (2016). Reprogramming macrophage orientation by microRNA 146b targeting transcription factor IRF5. EBioMedicine, 14, 83-96.
- Hu, J. K., Crampton, J. C., Locci, M., & Crotty, S. (2016). CRISPR-mediated Slamf1Δ/Δ Slamf5Δ/Δ Slamf6Δ/Δ triple gene disruption reveals NKT cell defects but not T follicular helper cell defects. PloS one, 11(5), e0156074.
- Smalley, E. (2016). CRISPR mouse model boom, rat model renaissance. Nature Biotechnology. 34, 893–894.
- Baker, M. (2014). Gene editing at CRISPR speed. Nature biotechnology, 32(4), 309-313.
TARGATT™ Site-Specific Knock-in Cell Lines
Book Chapters
- Chen-Tsai, R. Y. (2020). Integrase-Mediated Targeted Transgenics Through Pronuclear Microinjection. In Transgenic Mouse (pp. 35-46). Humana, New York, NY.
- Chen-Tsai, R. Y. (2019). Using TARGATT™ Technology to Generate Site-Specific Transgenic Mice. In Microinjection (pp. 71-86). Humana Press, New York, NY.
Master Cell Line
- Chi, X., Zheng, Q., Jiang, R., Chen-Tsai, R. Y., & Kong, L. J. (2019). A system for site-specific integration of transgenes in mammalian cells. PLOS ONE, 14(7), e0219842.
Description of the technology
- Zhu, F., Gamboa, M., Farruggio, A. P., Hippenmeyer, S., Tasic, B., Schüle, B., … Calos, M. P. (2014). DICE, an efficient system for iterative genomic editing in human pluripotent stem cells. Nucleic Acids Research, 42(5), e34. http://doi.org/10.1093/nar/gkt1290.
- Tasic, B., Hippenmeyer, S., Wang, C., Gamboa, M., Zong, H., Chen-Tsai, Y., & Luo, L. (2011). Site-specific integrase-mediated transgenesis in mice via pronuclear injection. Proceedings of the National Academy of Sciences of the United States of America, 108(19), 7902–7907. http://doi.org/10.1073/pnas.1019507108.
Commentary, comparison with other transgenic methods
- Rossant, J., Nutter, L. M., & Gertsenstein, M. (2011). Engineering the embryo. Proceedings of the National Academy of Sciences, 108(19), 7659-7660.
Tet inducible mice generated by TARGATT™
- Fan, X., Petitt, M., Gamboa, M., Huang, M., Dhal, S., Druzin, M. L., … Nayak, N. R. (2012). Transient, Inducible, Placenta-Specific Gene Expression in Mice. Endocrinology, 153(11), 5637–5644. http://doi.org/10.1210/en.2012-1556.
Advantage of Hipp11 (H11) locus
- Hippenmeyer, S., Youn, Y. H., Moon, H. M., Miyamichi, K., Zong, H., Wynshaw-Boris, A., & Luo, L. (2010). Genetic Mosaic Dissection of Lis1 and Ndel1 in Neuronal Migration. Neuron, 68(4), 695–709. http://doi.org/10.1016/j.neuron.2010.09.027.
Applications for mice generated by TARGATT™ (and cited/published articles)
- Carlson, H. L., & Stadler, H. S. (2019). Development and functional characterization of a lncRNA‐HIT conditional loss of function allele. genesis, e23351.
- Chande, S., Ho, B., Fetene, J., & Bergwitz, C. (2019). Transgenic mouse model for conditional expression of influenza hemagglutinin-tagged human SLC20A1/PIT1. PloS one, 14(10), e0223052. doi:10.1371/journal.pone.0223052
- Hu, Q., Ye, Y., Chan, L. C., Li, Y., Liang, K., Lin, A., … & Pan, Y. (2019). Oncogenic lncRNA downregulates cancer cell antigen presentation and intrinsic tumor suppression. Nature immunology, 1.
- Matharu, N., Rattanasopha, S., Tamura, S., Maliskova, L., Wang, Y., Bernard, A., … & Ahituv, N. (2018). CRISPR-mediated activation of a promoter or enhancer rescues obesity caused by haploinsufficiency. Science, eaau0629.
- Barrett, R. D., Laurent, S., Mallarino, R., Pfeifer, S. P., Xu, C. C., Foll, M., … & Hoekstra, H. E. (2018). The fitness consequences of genetic variation in wild populations of mice. bioRxiv, 383240.
- Ibrahim, L. A., Huang, J. J., Wang, S. Z., Kim, Y. J., Li, I., & Huizhong, W. (2018). Sparse Labeling and Neural Tracing in Brain Circuits by STARS Strategy: Revealing Morphological Development of Type II Spiral Ganglion Neurons. Cerebral Cortex, 1-14.
- Kumar, A., Dhar, S., Campanelli, G., Butt, N. A., Schallheim, J. M., Gomez, C. R., & Levenson, A. S. (2018). MTA 1 drives malignant progression and bone metastasis in prostate cancer. Molecular oncology.
- Jang, Y., Wang, C., Broun, A., Park, Y. K., Zhuang, L., Lee, J. E., … & Ge, K. (2018). H3. 3K4M destabilizes enhancer epigenomic writers MLL3/4 and impairs adipose tissue development. bioRxiv, 301986. doi:https://doi.org/10.1101/301986
- Tang, Y., Kwon, H., Neel, B. A., Kasher-Meron, M., Pessin, J., Yamada, E., & Pessin, J. E. (2018). The fructose-2, 6-bisphosphatase TIGAR suppresses NF-κB signaling by directly inhibiting the linear ubiquitin assembly complex LUBAC. Journal of Biological Chemistry, jbc-RA118.
- Chen, M., Geoffroy, C. G., Meves, J. M., Narang, A., Li, Y., Nguyen, M. T., … & Elzière, L. (2018). Leucine Zipper-Bearing Kinase Is a Critical Regulator of Astrocyte Reactivity in the Adult Mammalian CNS. Cell Reports, 22(13), 3587-3597.
- Kido, T., Sun, Z., & Lau, Y.-F. C. (2017). Aberrant activation of the human sex-determining gene in early embryonic development results in postnatal growth retardation and lethality in mice. Scientific Reports, 7, 4113. http://doi.org/10.1038/s41598-017-04117-6.
- Nouri, N., & Awatramani, R. (2017). A novel floor plate boundary defined by adjacent En1 and Dbx1 microdomains distinguishes midbrain dopamine and hypothalamic neurons. Development, 144(5), 916-927.
- Li, K., Wang, F., Cao, W. B., Lv, X. X., Hua, F., Cui, B., … & Yu, J. M. (2017). TRIB3 Promotes APL Progression through Stabilization of the Oncoprotein PML-RARα and Inhibition of p53-Mediated Senescence. Cancer Cell, 31(5), 697-710.
- Matharu, N., Rattanasopha, S., Maliskova, L., Wang, Y., Hardin, A., Vaisse, C., & Ahituv, N. (2017). Promoter or Enhancer Activation by CRISPRa Rescues Haploinsufficiency Caused Obesity. bioRxiv, 140426.
- Jiang, T., Kindt, K., & Wu, D. K. (2017). Transcription factor Emx2 controls stereociliary bundle orientation of sensory hair cells. eLife, 6, e23661.
- Booze, M. L., Hansen, J. M., & Vitiello, P. F. (2016). A Novel Mouse Model for the Identification of Thioredoxin-1 Protein Interactions. Free Radical Biology & Medicine, 99, 533–543. http://doi.org/10.1016/j.freeradbiomed.2016.09.013.
- Feng, D., Dai, S., Liu, F., Ohtake, Y., Zhou, Z., Wang, H., … & Hayat, U. (2016). Cre-inducible human CD59 mediates rapid cell ablation after intermedilysin administration. The Journal of clinical investigation, 126(6), 2321-2333.
- Sun, N., Yun, J., Liu, J., Malide, D., Liu, C., Rovira, I. I., … Finkel, T. (2015). Measuring in vivo mitophagy. Molecular Cell, 60(4), 685–696. http://doi.org/10.1016/j.molcel.2015.10.009.
- Devine, W. P., Wythe, J. D., George, M., Koshiba-Takeuchi, K., & Bruneau, B. G. (2014). Early patterning and specification of cardiac progenitors in gastrulating mesoderm. eLife, 3, e03848. http://doi.org/10.7554/eLife.03848.
- Fogg, P. C. M., Colloms, S., Rosser, S., Stark, M., & Smith, M. C. M. (2014). New Applications for Phage Integrases. Journal of Molecular Biology, 426(15), 2703–2716. http://doi.org/10.1016/j.jmb.2014.05.014.
- Chen-Tsai, R. Y., Jiang, R., Zhuang, L., Wu, J., Li, L., & Wu, J. (2014). Genome editing and animal models. Chinese science bulletin, 59(1), 1-6.
- Park, K.-E., Park, C.-H., Powell, A., Martin, J., Donovan, D. M., & Telugu, B. P. (2016). Targeted Gene Knockin in Porcine Somatic Cells Using CRISPR/Cas Ribonucleoproteins. International Journal of Molecular Sciences, 17(6), 810. http://doi.org/10.3390/ijms17060810.
- Guenther, C. A., Tasic, B., Luo, L., Bedell, M. A., & Kingsley, D. M. (2014). A molecular basis for classic blond hair color in Europeans. Nature Genetics, 46(7), 748–752. http://doi.org/10.1038/ng.2991.
- Villamizar, C. A. (2014). Characterization of the vascular pathology in the acta2 r258c mouse model and cerebrovascular characterization of the acta2 null mouse. UT GSBS Dissertations and These (Open Access). Paper 508 (2014)
MOUSE MODELS: Knock-in Mouse, Knockout Mouse Models
TARGATT™ Site Specific Knock-in Mouse
Book Chapters
- Chen-Tsai, R. Y. (2020). Integrase-Mediated Targeted Transgenics Through Pronuclear Microinjection. In Transgenic Mouse (pp. 35-46). Humana, New York, NY.
- Chen-Tsai, R. Y. (2019). Using TARGATT™ Technology to Generate Site-Specific Transgenic Mice. In Microinjection (pp. 71-86). Humana Press, New York, NY.
Master Cell Line
- Chi, X., Zheng, Q., Jiang, R., Chen-Tsai, R. Y., & Kong, L. J. (2019). A system for site-specific integration of transgenes in mammalian cells. PLOS ONE, 14(7), e0219842.
Description of the technology
- Zhu, F., Gamboa, M., Farruggio, A. P., Hippenmeyer, S., Tasic, B., Schüle, B., … Calos, M. P. (2014). DICE, an efficient system for iterative genomic editing in human pluripotent stem cells. Nucleic Acids Research, 42(5), e34. http://doi.org/10.1093/nar/gkt1290.
- Tasic, B., Hippenmeyer, S., Wang, C., Gamboa, M., Zong, H., Chen-Tsai, Y., & Luo, L. (2011). Site-specific integrase-mediated transgenesis in mice via pronuclear injection. Proceedings of the National Academy of Sciences of the United States of America, 108(19), 7902–7907. http://doi.org/10.1073/pnas.1019507108.
Commentary, comparison with other transgenic methods
- Rossant, J., Nutter, L. M., & Gertsenstein, M. (2011). Engineering the embryo. Proceedings of the National Academy of Sciences, 108(19), 7659-7660.
Tet inducible mice generated by TARGATT™
- Fan, X., Petitt, M., Gamboa, M., Huang, M., Dhal, S., Druzin, M. L., … Nayak, N. R. (2012). Transient, Inducible, Placenta-Specific Gene Expression in Mice. Endocrinology, 153(11), 5637–5644. http://doi.org/10.1210/en.2012-1556.
Advantage of Hipp11 (H11) locus
- Hippenmeyer, S., Youn, Y. H., Moon, H. M., Miyamichi, K., Zong, H., Wynshaw-Boris, A., & Luo, L. (2010). Genetic Mosaic Dissection of Lis1 and Ndel1 in Neuronal Migration. Neuron, 68(4), 695–709. http://doi.org/10.1016/j.neuron.2010.09.027.
Applications for mice generated by TARGATT™ (and cited/published articles
- Lindtner, S., Catta-Preta, R., Tian, H., Su-Feher, L., Price, J. D., Dickel, D. E., … & Pennacchio, L. A. (2019). Genomic Resolution of DLX-Orchestrated Transcriptional Circuits Driving Development of Forebrain GABAergic Neurons. Cell reports, 28(8), 2048-2063.
- Wang, T. A., Teo, C. F., Åkerblom, M., Chen, C., Tynan-La Fontaine, M., Greiner, V. J., … & Jan, L. Y. (2019). Thermoregulation via Temperature-Dependent PGD2 Production in Mouse Preoptic Area. Neuron, 103(2), 309-322.
- Clarke, B. A., Majumder, S., Zhu, H., Lee, Y. T., Kono, M., Li, C., … & Byrnes, C. (2019). The Ormdl genes regulate the sphingolipid synthesis pathway to ensure proper myelination and neurologic function in mice. eLife, 8.
- Carlson, H. L., & Stadler, H. S. (2019). Development and functional characterization of a lncRNA‐HIT conditional loss of function allele. genesis, e23351.
- Chande, S., Ho, B., Fetene, J., & Bergwitz, C. (2019). Transgenic mouse model for conditional expression of influenza hemagglutinin-tagged human SLC20A1/PIT1. PloS one, 14(10), e0223052. doi:10.1371/journal.pone.0223052
- Hu, Q., Ye, Y., Chan, L. C., Li, Y., Liang, K., Lin, A., … & Pan, Y. (2019). Oncogenic lncRNA downregulates cancer cell antigen presentation and intrinsic tumor suppression. Nature immunology, 1.
- Matharu, N., Rattanasopha, S., Tamura, S., Maliskova, L., Wang, Y., Bernard, A., … & Ahituv, N. (2018). CRISPR-mediated activation of a promoter or enhancer rescues obesity caused by haploinsufficiency. Science, eaau0629.
- Barrett, R. D., Laurent, S., Mallarino, R., Pfeifer, S. P., Xu, C. C., Foll, M., … & Hoekstra, H. E. (2018). The fitness consequences of genetic variation in wild populations of mice. bioRxiv, 383240.
- Ibrahim, L. A., Huang, J. J., Wang, S. Z., Kim, Y. J., Li, I., & Huizhong, W. (2018). Sparse Labeling and Neural Tracing in Brain Circuits by STARS Strategy: Revealing Morphological Development of Type II Spiral Ganglion Neurons. Cerebral Cortex, 1-14.
- Kumar, A., Dhar, S., Campanelli, G., Butt, N. A., Schallheim, J. M., Gomez, C. R., & Levenson, A. S. (2018). MTA 1 drives malignant progression and bone metastasis in prostate cancer. Molecular oncology.
- Jang, Y., Wang, C., Broun, A., Park, Y. K., Zhuang, L., Lee, J. E., … & Ge, K. (2018). H3. 3K4M destabilizes enhancer epigenomic writers MLL3/4 and impairs adipose tissue development. bioRxiv, 301986. doi:https://doi.org/10.1101/301986
- Tang, Y., Kwon, H., Neel, B. A., Kasher-Meron, M., Pessin, J., Yamada, E., & Pessin, J. E. (2018). The fructose-2, 6-bisphosphatase TIGAR suppresses NF-κB signaling by directly inhibiting the linear ubiquitin assembly complex LUBAC. Journal of Biological Chemistry, jbc-RA118.
- Chen, M., Geoffroy, C. G., Meves, J. M., Narang, A., Li, Y., Nguyen, M. T., … & Elzière, L. (2018). Leucine Zipper-Bearing Kinase Is a Critical Regulator of Astrocyte Reactivity in the Adult Mammalian CNS. Cell Reports, 22(13), 3587-3597.
- Kido, T., Sun, Z., & Lau, Y.-F. C. (2017). Aberrant activation of the human sex-determining gene in early embryonic development results in postnatal growth retardation and lethality in mice. Scientific Reports, 7, 4113. http://doi.org/10.1038/s41598-017-04117-6.
- Nouri, N., & Awatramani, R. (2017). A novel floor plate boundary defined by adjacent En1 and Dbx1 microdomains distinguishes midbrain dopamine and hypothalamic neurons. Development, 144(5), 916-927.
- Li, K., Wang, F., Cao, W. B., Lv, X. X., Hua, F., Cui, B., … & Yu, J. M. (2017). TRIB3 Promotes APL Progression through Stabilization of the Oncoprotein PML-RARα and Inhibition of p53-Mediated Senescence. Cancer Cell, 31(5), 697-710.
- Matharu, N., Rattanasopha, S., Maliskova, L., Wang, Y., Hardin, A., Vaisse, C., & Ahituv, N. (2017). Promoter or Enhancer Activation by CRISPRa Rescues Haploinsufficiency Caused Obesity. bioRxiv, 140426.
- Jiang, T., Kindt, K., & Wu, D. K. (2017). Transcription factor Emx2 controls stereociliary bundle orientation of sensory hair cells. eLife, 6, e23661.
- Booze, M. L., Hansen, J. M., & Vitiello, P. F. (2016). A Novel Mouse Model for the Identification of Thioredoxin-1 Protein Interactions. Free Radical Biology & Medicine, 99, 533–543. http://doi.org/10.1016/j.freeradbiomed.2016.09.013.
- Feng, D., Dai, S., Liu, F., Ohtake, Y., Zhou, Z., Wang, H., … & Hayat, U. (2016). Cre-inducible human CD59 mediates rapid cell ablation after intermedilysin administration. The Journal of clinical investigation, 126(6), 2321-2333.
- Sun, N., Yun, J., Liu, J., Malide, D., Liu, C., Rovira, I. I., … Finkel, T. (2015). Measuring in vivo mitophagy. Molecular Cell, 60(4), 685–696. http://doi.org/10.1016/j.molcel.2015.10.009.
- Devine, W. P., Wythe, J. D., George, M., Koshiba-Takeuchi, K., & Bruneau, B. G. (2014). Early patterning and specification of cardiac progenitors in gastrulating mesoderm. eLife, 3, e03848. http://doi.org/10.7554/eLife.03848.
- Fogg, P. C. M., Colloms, S., Rosser, S., Stark, M., & Smith, M. C. M. (2014). New Applications for Phage Integrases. Journal of Molecular Biology, 426(15), 2703–2716. http://doi.org/10.1016/j.jmb.2014.05.014.
- Chen-Tsai, R. Y., Jiang, R., Zhuang, L., Wu, J., Li, L., & Wu, J. (2014). Genome editing and animal models. Chinese science bulletin, 59(1), 1-6.
- Park, K.-E., Park, C.-H., Powell, A., Martin, J., Donovan, D. M., & Telugu, B. P. (2016). Targeted Gene Knockin in Porcine Somatic Cells Using CRISPR/Cas Ribonucleoproteins. International Journal of Molecular Sciences, 17(6), 810. http://doi.org/10.3390/ijms17060810.
- Guenther, C. A., Tasic, B., Luo, L., Bedell, M. A., & Kingsley, D. M. (2014). A molecular basis for classic blond hair color in Europeans. Nature Genetics, 46(7), 748–752. http://doi.org/10.1038/ng.2991.
- Villamizar, C. A. (2014). Characterization of the vascular pathology in the acta2 r258c mouse model and cerebrovascular characterization of the acta2 null mouse. UT GSBS Dissertations and These (Open Access). Paper 508 (2014)
CRISPR Knock-in, CRISPR Knockout Mouse
CRISPR Technology
- Smalley, E. (2016). CRISPR mouse model boom, rat model renaissance. Nature Biotechnology. 34, 893–894.
- Baker, M. (2014). Gene editing at CRISPR speed. Nature biotechnology, 32(4), 309-313.
CRISPR Knock-in H11 Locus in Pigs
- Ruan, J., Li, H., Xu, K., Wu, T., Wei, J., Zhou, R., … & Chen-Tsai, R. Y. (2015). Highly efficient CRISPR/Cas9-mediated transgene knockin at the H11 locus in pigs. Scientific reports, 5, 14253.
Knock-in, Knockout, Conditional Knock-out
- Park, J., Jung, E., Lee, S. H., & Chung, W. S. (2020). CDC50A dependent phosphatidylserine exposure induces inhibitory post-synapse elimination by microglia. bioRxiv.
- Ramachandra Rao, S., Fliesler, S. J., Kotla, P., Nguyen, M. N., & Pittler, S. J. (2020). Lack of Overt Retinal Degeneration in a K42E Dhdds Knock-In Mouse Model of RP59. Cells, 9(4), 896.
- Beurg, M., Barlow, A., Furness, D. N., & Fettiplace, R. (2019). A Tmc1 mutation reduces calcium permeability and expression of mechanoelectrical transduction channels in cochlear hair cells. Proceedings of the National Academy of Sciences, 116(41), 20743-20749.
- Goldring, A. C., Beurg, M., & Fettiplace, R. (2019). The contribution of TMC1 to adaptation of mechanoelectrical transduction channels in cochlear outer hair cells. The Journal of physiology.
- Hwang, S., He, Y., Xiang, X., Seo, W., Kim, S. J., Ma, J., … & Kunos, G. (2019). Interleukin‐22 ameliorates neutrophil‐driven nonalcoholic steatohepatitis through multiple targets. Hepatology https://doi.org/10.1002/hep.31031.
- Dumesic, P. A., Egan, D. F., Gut, P., Tran, M. T., Parisi, A., Chatterjee, N., … & Dou, F. (2019). An Evolutionarily Conserved uORF Regulates PGC1α and Oxidative Metabolism in Mice, Flies, and Bluefin Tuna. Cell metabolism.
- Liang, T., Zhang, H., Xu, Q., Wang, S., Qin, C., & Lu, Y. (2019). Mutant Dentin Sialophosphoprotein Causes Dentinogenesis Imperfecta. Journal of dental research, 0022034519854029.
- Qian, W., Miner, C. A., Ingle, H., Platt, D. J., Baldridge, M. T., & Miner, J. J. (2019). A human STAT1 gain-of-function mutation impairs CD8+ T cell responses against gammaherpesvirus-68. Journal of virology, JVI-00307.
- Kweon, S. M., Chen, Y., Moon, E., Kvederaviciutė, K., Klimasauskas, S., & Feldman, D. E. (2019). An Adversarial DNA N6-Methyladenine-Sensor Network Preserves Polycomb Silencing. Molecular Cell. https://doi.org/10.1016/j.molcel.2019.03.018
- Deng, F., He, S., Cui, S., Shi, Y., Tan, Y., Li, Z., … & Peng, L. (2019). A molecular targeted immunotherapeutic strategy for ulcerative colitis via dual-targeting nanoparticles delivering miR-146b to intestinal macrophages. Journal of Crohn’s and Colitis, 13(4), 482-494.
- Jo, Sungro, Tatiana L. Fonseca, Barbara MLC Bocco, Gustavo W. Fernandes, Elizabeth A. McAninch, Anaysa P. Bolin, Rodrigo R. Da Conceição et al. “Type 2 deiodinase polymorphism causes ER stress and hypothyroidism in the brain.” The Journal of clinical investigation 129, no. 1 (2019): 230-245.
- Langston, R. G., Rudenko, I. N., Kumaran, R., Hauser, D. N., Kaganovich, A., Ponce, L. B., … & Beilina, A. (2018). Differences in Stability, Activity and Mutation Effects Between Human and Mouse Leucine-Rich Repeat Kinase 2. Neurochemical research, 1-14.
- Amara, N., Tholen, M., & Bogyo, M. (2018). Chemical tools for selective activity profiling of endogenously expressed MMP-14 in multicellular models. ACS Chemical Biology. doi: 10.1021/acschembio.8b00562.
- Allocca, S., Ciano, M., Ciardulli, M. C., D’Ambrosio, C., Scaloni, A., Sarnataro, D., … & Bonatti, S. (2018). An αB-Crystallin Peptide Rescues Compartmentalization and Trafficking Response to Cu Overload of ATP7B-H1069Q, the Most Frequent Cause of Wilson Disease in the Caucasian Population. International journal of molecular sciences, 19(7).
- Peng, L., Zhang, H., Hao, Y., Xu, F., Yang, J., Zhang, R., … & Chen, C. (2016). Reprogramming macrophage orientation by microRNA 146b targeting transcription factor IRF5. EBioMedicine, 14, 83-96.
- Hu, J. K., Crampton, J. C., Locci, M., & Crotty, S. (2016). CRISPR-mediated Slamf1Δ/Δ Slamf5Δ/Δ Slamf6Δ/Δ triple gene disruption reveals NKT cell defects but not T follicular helper cell defects. PloS one, 11(5), e0156074.
- Besschetnova, T. Y., Ichimura, T., Katebi, N., Croix, B. S., Bonventre, J. V., & Olsen, B. R. (2015). Regulatory mechanisms of anthrax toxin receptor 1-dependent vascular and connective tissue homeostasis. Matrix Biology, 42, 56-73.
- McKenzie, C. W., Craige, B., Kroeger, T. V., Finn, R., Wyatt, T. A., Sisson, J. H., … & Lee, L. (2015). CFAP54 is required for proper ciliary motility and assembly of the central pair apparatus in mice. Molecular biology of the cell, 26(18), 3140-3149.
- Bishop, K. A., Harrington, A., Kouranova, E., Weinstein, E. J., Rosen, C. J., Cui, X., & Liaw, L. (2016). CRISPR/Cas9-mediated insertion of loxP sites in the mouse Dock7 gene provides an effective alternative to use of targeted embryonic stem cells. G3: Genes, Genomes, Genetics, 6(7), 2051-2061.
Homologous Recombination Conditional Knockout Mouse
- Zhao, M., Tao, F., Venkatraman, A., Li, Z., Smith, S. E., Unruh, J., … & Marshall, H. (2019). N-Cadherin-Expressing Bone and Marrow Stromal Progenitor Cells Maintain Reserve Hematopoietic Stem Cells. Cell reports, 26(3), 652-669.
- Li, C., Zheng, Z., Ha, P., Chen, X., Jiang, W., Sun, S., … & Chen, E. C. (2018). Neurexin Superfamily Cell Membrane Receptor Contactin‐Associated Protein Like‐4 (Cntnap4) is Involved in Neural EGFL Like 1 (Nell‐1)‐responsive Osteogenesis. Journal of Bone and Mineral Research https://doi.org/10.1002/jbmr.3524.
- Geraets, R. D., Langin, L. M., Cain, J. T., Parker, C. M., Beraldi, R., Kovacs, A. D., … & Pearce, D. A. (2017). A tailored mouse model of CLN2 disease: A nonsense mutant for testing personalized therapies. PloS one, 12(5), e0176526.
- Miller, J. N., Kovács, A. D., & Pearce, D. A. (2015). The novel Cln1R151Xmouse model of infantile neuronal ceroid lipofuscinosis (INCL) for testing nonsense suppression therapy. Human Molecular Genetics, 24(1), 185–196. http://doi.org/10.1093/hmg/ddu428.
RAT MODELS: Knock-in Rat Models, Conditional Knockout Rat
TARGATT™ Site-Specific Knock-in Rat H11
Book Chapters
- Chen-Tsai, R. Y. (2020). Integrase-Mediated Targeted Transgenics Through Pronuclear Microinjection. In Transgenic Mouse (pp. 35-46). Humana, New York, NY.
- Chen-Tsai, R. Y. (2019). Using TARGATT™ Technology to Generate Site-Specific Transgenic Mice. In Microinjection (pp. 71-86). Humana Press, New York, NY.
Master Cell Line
- Chi, X., Zheng, Q., Jiang, R., Chen-Tsai, R. Y., & Kong, L. J. (2019). A system for site-specific integration of transgenes in mammalian cells. PLOS ONE, 14(7), e0219842.
Description of the technology
- POSTER. 13th Transgenic Technology Meeting being held in Prague, Czech Republic. March 20-23, 2016
- Zhu, F., Gamboa, M., Farruggio, A. P., Hippenmeyer, S., Tasic, B., Schüle, B., … Calos, M. P. (2014). DICE, an efficient system for iterative genomic editing in human pluripotent stem cells. Nucleic Acids Research, 42(5), e34. http://doi.org/10.1093/nar/gkt1290.
- Tasic, B., Hippenmeyer, S., Wang, C., Gamboa, M., Zong, H., Chen-Tsai, Y., & Luo, L. (2011). Site-specific integrase-mediated transgenesis in mice via pronuclear injection. Proceedings of the National Academy of Sciences of the United States of America, 108(19), 7902–7907. http://doi.org/10.1073/pnas.1019507108.
Commentary, comparison with other transgenic methods
- Rossant, J., Nutter, L. M., & Gertsenstein, M. (2011). Engineering the embryo. Proceedings of the National Academy of Sciences, 108(19), 7659-7660.
Tet inducible mice generated by TARGATT™
- Fan, X., Petitt, M., Gamboa, M., Huang, M., Dhal, S., Druzin, M. L., … Nayak, N. R. (2012). Transient, Inducible, Placenta-Specific Gene Expression in Mice. Endocrinology, 153(11), 5637–5644. http://doi.org/10.1210/en.2012-1556.
Advantage of Hipp11 (H11) locus
- Hippenmeyer, S., Youn, Y. H., Moon, H. M., Miyamichi, K., Zong, H., Wynshaw-Boris, A., & Luo, L. (2010). Genetic Mosaic Dissection of Lis1 and Ndel1 in Neuronal Migration. Neuron, 68(4), 695–709. http://doi.org/10.1016/j.neuron.2010.09.027.
Applications for mice generated by TARGATT™ (and cited/published articles)
- Lindtner, S., Catta-Preta, R., Tian, H., Su-Feher, L., Price, J. D., Dickel, D. E., … & Pennacchio, L. A. (2019). Genomic Resolution of DLX-Orchestrated Transcriptional Circuits Driving Development of Forebrain GABAergic Neurons. Cell reports, 28(8), 2048-2063.
- Wang, T. A., Teo, C. F., Åkerblom, M., Chen, C., Tynan-La Fontaine, M., Greiner, V. J., … & Jan, L. Y. (2019). Thermoregulation via Temperature-Dependent PGD2 Production in Mouse Preoptic Area. Neuron, 103(2), 309-322.
- Clarke, B. A., Majumder, S., Zhu, H., Lee, Y. T., Kono, M., Li, C., … & Byrnes, C. (2019). The Ormdl genes regulate the sphingolipid synthesis pathway to ensure proper myelination and neurologic function in mice. eLife, 8.
- Carlson, H. L., & Stadler, H. S. (2019). Development and functional characterization of a lncRNA‐HIT conditional loss of function allele. genesis, e23351.
- Chande, S., Ho, B., Fetene, J., & Bergwitz, C. (2019). Transgenic mouse model for conditional expression of influenza hemagglutinin-tagged human SLC20A1/PIT1. PloS one, 14(10), e0223052. doi:10.1371/journal.pone.0223052
- Hu, Q., Ye, Y., Chan, L. C., Li, Y., Liang, K., Lin, A., … & Pan, Y. (2019). Oncogenic lncRNA downregulates cancer cell antigen presentation and intrinsic tumor suppression. Nature immunology, 1.
- Matharu, N., Rattanasopha, S., Tamura, S., Maliskova, L., Wang, Y., Bernard, A., … & Ahituv, N. (2018). CRISPR-mediated activation of a promoter or enhancer rescues obesity caused by haploinsufficiency. Science, eaau0629.
- Barrett, R. D., Laurent, S., Mallarino, R., Pfeifer, S. P., Xu, C. C., Foll, M., … & Hoekstra, H. E. (2018). The fitness consequences of genetic variation in wild populations of mice. bioRxiv, 383240.
- Ibrahim, L. A., Huang, J. J., Wang, S. Z., Kim, Y. J., Li, I., & Huizhong, W. (2018). Sparse Labeling and Neural Tracing in Brain Circuits by STARS Strategy: Revealing Morphological Development of Type II Spiral Ganglion Neurons. Cerebral Cortex, 1-14.
- Kumar, A., Dhar, S., Campanelli, G., Butt, N. A., Schallheim, J. M., Gomez, C. R., & Levenson, A. S. (2018). MTA 1 drives malignant progression and bone metastasis in prostate cancer. Molecular oncology.
- Jang, Y., Wang, C., Broun, A., Park, Y. K., Zhuang, L., Lee, J. E., … & Ge, K. (2018). H3. 3K4M destabilizes enhancer epigenomic writers MLL3/4 and impairs adipose tissue development. bioRxiv, 301986. doi:https://doi.org/10.1101/301986
- Tang, Y., Kwon, H., Neel, B. A., Kasher-Meron, M., Pessin, J., Yamada, E., & Pessin, J. E. (2018). The fructose-2, 6-bisphosphatase TIGAR suppresses NF-κB signaling by directly inhibiting the linear ubiquitin assembly complex LUBAC. Journal of Biological Chemistry, jbc-RA118.
- Chen, M., Geoffroy, C. G., Meves, J. M., Narang, A., Li, Y., Nguyen, M. T., … & Elzière, L. (2018). Leucine Zipper-Bearing Kinase Is a Critical Regulator of Astrocyte Reactivity in the Adult Mammalian CNS. Cell Reports, 22(13), 3587-3597.
- Kido, T., Sun, Z., & Lau, Y.-F. C. (2017). Aberrant activation of the human sex-determining gene in early embryonic development results in postnatal growth retardation and lethality in mice. Scientific Reports, 7, 4113. http://doi.org/10.1038/s41598-017-04117-6.
- Nouri, N., & Awatramani, R. (2017). A novel floor plate boundary defined by adjacent En1 and Dbx1 microdomains distinguishes midbrain dopamine and hypothalamic neurons. Development, 144(5), 916-927.
- Li, K., Wang, F., Cao, W. B., Lv, X. X., Hua, F., Cui, B., … & Yu, J. M. (2017). TRIB3 Promotes APL Progression through Stabilization of the Oncoprotein PML-RARα and Inhibition of p53-Mediated Senescence. Cancer Cell, 31(5), 697-710.
- Matharu, N., Rattanasopha, S., Maliskova, L., Wang, Y., Hardin, A., Vaisse, C., & Ahituv, N. (2017). Promoter or Enhancer Activation by CRISPRa Rescues Haploinsufficiency Caused Obesity. bioRxiv, 140426.
- Jiang, T., Kindt, K., & Wu, D. K. (2017). Transcription factor Emx2 controls stereociliary bundle orientation of sensory hair cells. eLife, 6, e23661.
- Booze, M. L., Hansen, J. M., & Vitiello, P. F. (2016). A Novel Mouse Model for the Identification of Thioredoxin-1 Protein Interactions. Free Radical Biology & Medicine, 99, 533–543. http://doi.org/10.1016/j.freeradbiomed.2016.09.013.
- Feng, D., Dai, S., Liu, F., Ohtake, Y., Zhou, Z., Wang, H., … & Hayat, U. (2016). Cre-inducible human CD59 mediates rapid cell ablation after intermedilysin administration. The Journal of clinical investigation, 126(6), 2321-2333.
- Sun, N., Yun, J., Liu, J., Malide, D., Liu, C., Rovira, I. I., … Finkel, T. (2015). Measuring in vivo mitophagy. Molecular Cell, 60(4), 685–696. http://doi.org/10.1016/j.molcel.2015.10.009.
- Devine, W. P., Wythe, J. D., George, M., Koshiba-Takeuchi, K., & Bruneau, B. G. (2014). Early patterning and specification of cardiac progenitors in gastrulating mesoderm. eLife, 3, e03848. http://doi.org/10.7554/eLife.03848.
- Fogg, P. C. M., Colloms, S., Rosser, S., Stark, M., & Smith, M. C. M. (2014). New Applications for Phage Integrases. Journal of Molecular Biology, 426(15), 2703–2716. http://doi.org/10.1016/j.jmb.2014.05.014.
- Chen-Tsai, R. Y., Jiang, R., Zhuang, L., Wu, J., Li, L., & Wu, J. (2014). Genome editing and animal models. Chinese science bulletin, 59(1), 1-6.
- Park, K.-E., Park, C.-H., Powell, A., Martin, J., Donovan, D. M., & Telugu, B. P. (2016). Targeted Gene Knockin in Porcine Somatic Cells Using CRISPR/Cas Ribonucleoproteins. International Journal of Molecular Sciences, 17(6), 810. http://doi.org/10.3390/ijms17060810.
- Guenther, C. A., Tasic, B., Luo, L., Bedell, M. A., & Kingsley, D. M. (2014). A molecular basis for classic blond hair color in Europeans. Nature Genetics, 46(7), 748–752. http://doi.org/10.1038/ng.2991.
- Villamizar, C. A. (2014). Characterization of the vascular pathology in the acta2 r258c mouse model and cerebrovascular characterization of the acta2 null mouse. UT GSBS Dissertations and These (Open Access). Paper 508 (2014)
CRISPR Knock-In, Knock-Out, Conditional Knock-Out Rat
CRISPR Technology
- Smalley, E. (2016). CRISPR mouse model boom, rat model renaissance. Nature Biotechnology. 34, 893–894.
- Baker, M. (2014). Gene editing at CRISPR speed. Nature biotechnology, 32(4), 309-313.
CRISPR Knock-in H11 Locus in Pigs
- Ruan, J., Li, H., Xu, K., Wu, T., Wei, J., Zhou, R., … & Chen-Tsai, R. Y. (2015). Highly efficient CRISPR/Cas9-mediated transgene knockin at the H11 locus in pigs. Scientific reports, 5, 14253.
Others
- Park, J., Jung, E., Lee, S. H., & Chung, W. S. (2020). CDC50A dependent phosphatidylserine exposure induces inhibitory post-synapse elimination by microglia. bioRxiv.
- Ramachandra Rao, S., Fliesler, S. J., Kotla, P., Nguyen, M. N., & Pittler, S. J. (2020). Lack of Overt Retinal Degeneration in a K42E Dhdds Knock-In Mouse Model of RP59. Cells, 9(4), 896.
- Beurg, M., Barlow, A., Furness, D. N., & Fettiplace, R. (2019). A Tmc1 mutation reduces calcium permeability and expression of mechanoelectrical transduction channels in cochlear hair cells. Proceedings of the National Academy of Sciences, 116(41), 20743-20749.
- Goldring, A. C., Beurg, M., & Fettiplace, R. (2019). The contribution of TMC1 to adaptation of mechanoelectrical transduction channels in cochlear outer hair cells. The Journal of physiology.
- Hwang, S., He, Y., Xiang, X., Seo, W., Kim, S. J., Ma, J., … & Kunos, G. (2019). Interleukin‐22 ameliorates neutrophil‐driven nonalcoholic steatohepatitis through multiple targets. Hepatology https://doi.org/10.1002/hep.31031.
- Dumesic, P. A., Egan, D. F., Gut, P., Tran, M. T., Parisi, A., Chatterjee, N., … & Dou, F. (2019). An Evolutionarily Conserved uORF Regulates PGC1α and Oxidative Metabolism in Mice, Flies, and Bluefin Tuna. Cell metabolism.
- Liang, T., Zhang, H., Xu, Q., Wang, S., Qin, C., & Lu, Y. (2019). Mutant Dentin Sialophosphoprotein Causes Dentinogenesis Imperfecta. Journal of dental research, 0022034519854029.
- Qian, W., Miner, C. A., Ingle, H., Platt, D. J., Baldridge, M. T., & Miner, J. J. (2019). A human STAT1 gain-of-function mutation impairs CD8+ T cell responses against gammaherpesvirus-68. Journal of virology, JVI-00307.
- Kweon, S. M., Chen, Y., Moon, E., Kvederaviciutė, K., Klimasauskas, S., & Feldman, D. E. (2019). An Adversarial DNA N6-Methyladenine-Sensor Network Preserves Polycomb Silencing. Molecular Cell. https://doi.org/10.1016/j.molcel.2019.03.018
- Deng, F., He, S., Cui, S., Shi, Y., Tan, Y., Li, Z., … & Peng, L. (2019). A molecular targeted immunotherapeutic strategy for ulcerative colitis via dual-targeting nanoparticles delivering miR-146b to intestinal macrophages. Journal of Crohn’s and Colitis, 13(4), 482-494.
- Jo, Sungro, Tatiana L. Fonseca, Barbara MLC Bocco, Gustavo W. Fernandes, Elizabeth A. McAninch, Anaysa P. Bolin, Rodrigo R. Da Conceição et al. “Type 2 deiodinase polymorphism causes ER stress and hypothyroidism in the brain.” The Journal of clinical investigation 129, no. 1 (2019): 230-245.
- Langston, R. G., Rudenko, I. N., Kumaran, R., Hauser, D. N., Kaganovich, A., Ponce, L. B., … & Beilina, A. (2018). Differences in Stability, Activity and Mutation Effects Between Human and Mouse Leucine-Rich Repeat Kinase 2. Neurochemical research, 1-14.
- Amara, N., Tholen, M., & Bogyo, M. (2018). Chemical tools for selective activity profiling of endogenously expressed MMP-14 in multicellular models. ACS Chemical Biology. doi: 10.1021/acschembio.8b00562.
- Allocca, S., Ciano, M., Ciardulli, M. C., D’Ambrosio, C., Scaloni, A., Sarnataro, D., … & Bonatti, S. (2018). An αB-Crystallin Peptide Rescues Compartmentalization and Trafficking Response to Cu Overload of ATP7B-H1069Q, the Most Frequent Cause of Wilson Disease in the Caucasian Population. International journal of molecular sciences, 19(7).
- Peng, L., Zhang, H., Hao, Y., Xu, F., Yang, J., Zhang, R., … & Chen, C. (2016). Reprogramming macrophage orientation by microRNA 146b targeting transcription factor IRF5. EBioMedicine, 14, 83-96.
- Hu, J. K., Crampton, J. C., Locci, M., & Crotty, S. (2016). CRISPR-mediated Slamf1Δ/Δ Slamf5Δ/Δ Slamf6Δ/Δ triple gene disruption reveals NKT cell defects but not T follicular helper cell defects. PloS one, 11(5), e0156074.
- Besschetnova, T. Y., Ichimura, T., Katebi, N., Croix, B. S., Bonventre, J. V., & Olsen, B. R. (2015). Regulatory mechanisms of anthrax toxin receptor 1-dependent vascular and connective tissue homeostasis. Matrix Biology, 42, 56-73.
- McKenzie, C. W., Craige, B., Kroeger, T. V., Finn, R., Wyatt, T. A., Sisson, J. H., … & Lee, L. (2015). CFAP54 is required for proper ciliary motility and assembly of the central pair apparatus in mice. Molecular biology of the cell, 26(18), 3140-3149.
- Bishop, K. A., Harrington, A., Kouranova, E., Weinstein, E. J., Rosen, C. J., Cui, X., & Liaw, L. (2016). CRISPR/Cas9-mediated insertion of loxP sites in the mouse Dock7 gene provides an effective alternative to use of targeted embryonic stem cells. G3: Genes, Genomes, Genetics, 6(7), 2051-2061.
iPSC services
iPSC Generation
- Ilic, D. (2019). Latest developments in the field of stem cell research and regenerative medicine compiled from publicly available information and press releases from nonacademic institutions in October 2018. Regenerative medicine, 14(2), 85-92.
- Allende, M. L., Cook, E. K., Larman, B. C., Nugent, A., Brady, J. M., Golebiowski, D., … & Proia, R. L. (2018). Cerebral organoids derived from Sandhoff disease induced pluripotent stem cells exhibit impaired neurodifferentiation. Journal of Lipid Research, jlr-M081323.
- Field, A. R., Jacobs, F. M., Fiddes, I. T., Phillips, A. P., Reyes-Ortiz, A. M., LaMontagne, E., … & Hauessler, M. (2019). Structurally Conserved Primate LncRNAs Are Transiently Expressed during Human Cortical Differentiation and Influence Cell-Type-Specific Genes. Stem cell reports.
Safe Harbor Locus Master iPSC Generation with TARGATT™
- Karow, M., Chavez, C. L., Farruggio, A. P., Geisinger, J. M., Keravala, A., Jung, W. E., … & Calos, M. P. (2011). Site‐Specific Recombinase Strategy to Create Induced Pluripotent Stem Cells Efficiently with Plasmid DNA. Stem Cells, 29(11), 1696-1704.
- Zhu, F., Gamboa, M., Farruggio, A. P., Hippenmeyer, S., Tasic, B., Schüle, B., … & Calos, M. P. (2013). DICE, an efficient system for iterative genomic editing in human pluripotent stem cells. Nucleic acids research, 42(5), e34-e34.
iPSC Disease Modeling
- Ilic, D. (2019). Latest developments in the field of stem cell research and regenerative medicine compiled from publicly available information and press releases from nonacademic institutions in October 2018. Regenerative medicine, 14(2), 85-92.
- Simkin, D., Searl, T. J., Piyevsky, B. N., Forrest, M., Williams, L. A., Joshi, V., … & Penzes, P. (2019). Impaired M-current in KCNQ2 Encephalopathy Evokes Dyshomeostatic Modulation of Excitability. bioRxiv, 538371. https://doi.org/10.1101/538371
- Jang, Y., Choi, J., Park, N., Kang, J., Kim, M., Kim, Y., & Ju, J. H. (2019). Development of immunocompatible pluripotent stem cells via CRISPR-based human leukocyte antigen engineering. Experimental & Molecular Medicine, 51(1), 3.
- Lizarraga, S. B., Maguire, A. M., Ma, L., van Dyck, L. I., Wu, Q., Nagda, D., … & Cowen, M. H. (2018). Human neurons from Christianson syndrome iPSCs reveal allele-specific responses to rescue strategies. bioRxiv, 444232.
- Tanaka, H., Kondo, K., Chen, X., Homma, H., Tagawa, K., Kerever, A., … & Fujita, K. (2018). The intellectual disability gene PQBP1 rescues Alzheimer’s disease pathology. Molecular Psychiatry, 1.
- Selvan, N., George, S., Serajee, F. J., Shaw, M., Hobson, L., Kalscheuer, V. M., … & Schwartz, C. E. (2018). O-GlcNAc transferase missense mutations linked to X-linked intellectual disability deregulate genes involved in cell fate determination and signaling. Journal of Biological Chemistry, jbc-RA118.
- Chai, S., Wan, X., Ramirez-Navarro, A., Tesar, P. J., Kaufman, E. S., Ficker, E., … & Deschênes, I. (2018). Physiological genomics identifies genetic modifiers of long QT syndrome type 2 severity. The Journal of clinical investigation, 128(3).
- Seigel, G. M., et al. (2014). Comparative Analysis of ABCG2+ Stem-Like Retinoblastoma Cells and Induced Pluripotent Stem Cells as Three-Dimensional Aggregates. Investigative Ophthalmology & Visual Science, 55(13), 3068-3068.
- Comley, J. (2016). CRISPR/Cas9 – transforming gene editing in drug discovery labs. Drug Discovery Weekly. Fall 2016; 33-48.
Teratoma Analysis, iPSC Characterization
Teratoma Formation Assay (Published reports of ASC’s Teratoma Formation Analysis Reports can be found in the Certificate of Analysis “Induced Pluripotent Stem Cells (iPSCs)” by Coriell Institute for Medical Research.)
- DIABETES MELLITUS, JUVENILE-ONSET INSULIN-DEPENDENT; IDDM (Page 8-12)
- HUNTINGTON DISEASE; HD (Page 8-13)
- MUSCULAR DYSTROPHY, BECKER TYPE; BMD (Page 8-12)
- SEVERE COMBINED IMMUNODEFICIENCY, AUTOSOMAL RECESSIVE, T CELL-NEGATIVE, B CELL-NEGATIVE, NK CELL-NEGATIVE, DUE TO ADENOSINE DEAMINASE DEFICIENCY (Page 8-12)
- SPINAL MUSCULAR ATROPHY I; SMA1 (Page 8-15)
References (*cited/published articles)
- Ou, J., Ball, J. M., Luan, Y., Zhao, T., Miyagishima, K. J., Xu, Y., … & Mallon, B. S. (2018). iPSCs from a Hibernator Provide a Platform for Studying Cold Adaptation and Its Potential Medical Applications. Cell, 173(4), 851-863. https://doi.org/10.1016/j.cell.2018.03.010
- Teves, S. S., An, L., Bhargava-Shah, A., Xie, L., Darzacq, X., & Tjian, R. (2018). A stable mode of bookmarking by TBP recruits RNA Polymerase II to mitotic chromosomes. bioRxiv, 257451. DOI: 10.1101/257451
- Hansen, A. S., Pustova, I., Cattoglio, C., Tjian, R., & Darzacq, X. (2017). CTCF and cohesin regulate chromatin loop stability with distinct dynamics. Elife, 6.
- Vermilyea, S. C., Guthrie, S., Meyer, M., Smuga-Otto, K., Braun, K., Howden, S., … & Golos, T. G. (2017). Induced Pluripotent Stem Cell-Derived Dopaminergic Neurons from Adult Common Marmoset Fibroblasts. Stem cells and development, 26(17), 1225-1235. https://doi.org/10.1089/scd.2017.0069.
- Teves, S. S., An, L., Hansen, A. S., Xie, L., Darzacq, X., & Tjian, R. (2016). A dynamic mode of mitotic bookmarking by transcription factors. Elife, 5.
- Laskowski, T. J., Van Caeneghem, Y., Pourebrahim, R., Ma, C., Ni, Z., Garate, Z., … & Segovia, J. C. (2016). Gene correction of iPSCs from a Wiskott-Aldrich syndrome patient normalizes the lymphoid developmental and functional defects. Stem cell reports, 7(2), 139-148.
- Boza-Morán, M. G., Martínez-Hernández, R., Bernal, S., Wanisch, K., Also-Rallo, E., Le Heron, A., … & Tizzano, E. F. (2015). Decay in survival motor neuron and plastin 3 levels during differentiation of iPSC-derived human motor neurons. Scientific reports, 5, 11696.
- Romero, I. G., Pavlovic, B. J., Hernando-Herraez, I., Zhou, X., Ward, M. C., Banovich, N. E., … & Chavarria, C. I. (2015). A panel of induced pluripotent stem cells from chimpanzees: a resource for comparative functional genomics. Elife, 4.
- Cheung, H. S., Pelaez, D., & Huang, C. C. (2015). U.S. Patent Application No. 14/382,287.
- Chakravarti, D., Su, X., Cho, M. S., Bui, N. H. B., Coarfa, C., Venkatanarayan, A., … & Leung, M. L. (2014). Induced multipotency in adult keratinocytes through down-regulation of ΔNp63 or DGCR8. Proceedings of the National Academy of Sciences, 111(5), E572-E581.
- Lee, J., Kim, Y., Yi, H., Diecke, S., Kim, J., Jung, H., … & Park, S. H. (2014). Generation of disease-specific induced pluripotent stem cells from patients with rheumatoid arthritis and osteoarthritis. Arthritis research & therapy, 16(1), R41.
- Quang, T., Marquez, M., Blanco, G., & Zhao, Y. (2014). Dosage and cell line dependent inhibitory effect of bFGF supplement in human pluripotent stem cell culture on inactivated human mesenchymal stem cells. PloS one, 9(1), e86031.
- Jumabay, M., Abdmaulen, R., Ly, A., Cubberly, M. R., Shahmirian, L. J., Heydarkhan-Hagvall, S., … & Boström, K. I. (2014). Pluripotent stem cells derived from mouse and human white mature adipocytes. Stem cells translational medicine, 3(2), 161-171.
- Sanders, L. H., Laganière, J., Cooper, O., Mak, S. K., Vu, B. J., Huang, Y. A., … & Langston, J. W. (2014). LRRK2 mutations cause mitochondrial DNA damage in iPSC-derived neural cells from Parkinson’s disease patients: reversal by gene correction. Neurobiology of disease, 62, 381-386.
- Sun, N., & Zhao, H. (2014). Seamless correction of the sickle cell disease mutation of the HBB gene in human induced pluripotent stem cells using TALENs. Biotechnology and bioengineering, 111(5), 1048-1053.
- Lee, P., Martin, N. T., Nakamura, K., Azghadi, S., Amiri, M., Ben-David, U., … & Lowry, W. E. (2013). SMRT compounds abrogate cellular phenotypes of ataxia telangiectasia in neural derivatives of patient-specific hiPSCs. Nature communications, 4, 1824.
- Buccini, S. M. (2013). Doctoral dissertation, University of Cincinnati.
- Pelaez, D., Huang, C. Y. C., & Cheung, H. S. (2013). Isolation of pluripotent neural crest-derived stem cells from adult human tissues by connexin-43 enrichment. Stem cells and development, 22(21), 2906-2914.
- Cassidy, L., Choi, M., Meyer, J., Chang, R., & Seigel, G. M. (2013). Immunoreactivity of Pluripotent Markers SSEA-5 and L1CAM in Human Tumors, Teratomas, and Induced Pluripotent Stem Cells. Journal of Biomarkers, 2013, 960862. http://doi.org/10.1155/2013/960862.
- Cooper, O., Seo, H., Andrabi, S., Guardia-Laguarta, C., Graziotto, J., Sundberg, M., … Isacson, O. (2012). Familial Parkinson’s disease iPSCs show cellular deficits in mitochondrial responses that can be pharmacologically rescued. Science Translational Medicine, 4(141), 141ra90. http://doi.org/10.1126/scitranslmed.3003985.
- Zheng, Z., Jian, J., Zhang, X., Zara, J. N., Yin, W., Chiang, M., … & Soo, C. (2012). Reprogramming of human fibroblasts into multipotent cells with a single ECM proteoglycan, fibromodulin. Biomaterials, 33(24), 5821-5831.
- Almeida, S., Zhang, Z., Coppola, G., Mao, W., Futai, K., Karydas, A., … & Sena-Esteves, M. (2012). Induced pluripotent stem cell models of progranulin-deficient frontotemporal dementia uncover specific reversible neuronal defects. Cell reports, 2(4), 789-798.
- Zhang, W. Y., de Almeida, P. E., & Wu, J. C. (2012). Teratoma formation: A tool for monitoring pluripotency in stem cell research. StemBook.
- Jing, L., Christoforou, N., Leong, K. W., Setton, L. A., & Chen, J. (2012). Differentiation potential of human induced pluripotent stem cells (iPSCs) to nucleus pulposus-like cells in vitro. Global Spine Journal, 2(1_suppl), s-0032.
- Valamehr, B., Abujarour, R., Robinson, M., Le, T., Robbins, D., Shoemaker, D., & Flynn, P. (2012). A novel platform to enable the high-throughput derivation and characterization of feeder-free human iPSCs. Scientific reports, 2, 213.
- Chen, K. G., Mallon, B. S., Hamilton, R. S., Kozhich, O. A., Park, K., Hoeppner, D. J., … & McKay, R. D. (2012). Non-colony type monolayer culture of human embryonic stem cells. Stem cell research, 9(3), 237-248.
- Telugu, B. P. V. L., Ezashi, T., Sinha, S., Alexenko, A. P., Spate, L., Prather, R. S., & Roberts, R. M. (2011). Leukemia Inhibitory Factor (LIF)-dependent, Pluripotent Stem Cells Established from Inner Cell Mass of Porcine Embryos. The Journal of Biological Chemistry, 286(33), 28948–28953. http://doi.org/10.1074/jbc.M111.229468.
- Deleidi, M., Hargus, G., Hallett, P., Osborn, T., & Isacson, O. (2011). Development of histocompatible primate induced pluripotent stem cells for neural transplantation. Stem Cells (Dayton, Ohio), 29(7), 1052–1063. http://doi.org/10.1002/stem.662
Karyotyping (*cited/published articles)
- Zhao, L., Teklemariam, T., & Hantash, B. M. (2014). Heterelogous expression of mutated HLA-G decreases immunogenicity of human embryonic stem cells and their epidermal derivatives. Stem cell research, 13(2), 342-354.
- Sun, N., & Zhao, H. (2014). Seamless correction of the sickle cell disease mutation of the HBB gene in human induced pluripotent stem cells using TALENs. Biotechnology and bioengineering, 111(5), 1048-1053.
- An, M. C., Zhang, N., Scott, G., Montoro, D., Wittkop, T., Mooney, S., … & Ellerby, L. M. (2012). Genetic correction of Huntington’s disease phenotypes in induced pluripotent stem cells. Cell stem cell, 11(2), 253-263.
- Zheng, Z., Jian, J., Zhang, X., Zara, J. N., Yin, W., Chiang, M., … & Soo, C. (2012). Reprogramming of human fibroblasts into multipotent cells with a single ECM proteoglycan, fibromodulin. Biomaterials, 33(24), 5821-5831.
DNA Cloning/ Virus Packaging Service
Vector Design/ Cloning Service (*cited/published articles)
- Hu, Q., Li, C., Wang, S., Li, Y., Wen, B., Zhang, Y., … & Nguyen, T. K. (2019). LncRNAs-directed PTEN enzymatic switch governs epithelial–mesenchymal transition. Cell research, 1.
- Besschetnova, T. Y., Ichimura, T., Katebi, N., Croix, B. S., Bonventre, J. V., & Olsen, B. R. (2015). Regulatory mechanisms of anthrax toxin receptor 1-dependent vascular and connective tissue homeostasis. Matrix Biology, 42, 56-73.
Lentivirus Packaging
- Mathew, D. J. (2014). Characterization of interleukin-1 beta 2, a novel interleukin-1 expressed by the early pig conceptus during establishment of pregnancy. Doctoral dissertation, University of Missouri-Columbia.
Custom (CRO) Services
- Mace, E. M., Paust, S., Conte, M. I., Baxley, R. M., Schmit, M., Mukherjee, M., … & Akdemir, Z. C. (2019). Human NK cell deficiency as a result of biallelic mutations in MCM10. bioRxiv, 825554.
- Chen, H., Shi, M., Gilam, A., Zheng, Q., Zhang, Y., Afrikanova, I., … & Chen-Tsai, R. Y. (2019). Hemophilia A ameliorated in mice by CRISPR-based in vivo genome editing of human Factor VIII. Scientific reports, 9(1), 1-15.
- Baskfield, A., Li, R., Beers, J., Zou, J., Liu, C., & Zheng, W. (2019). Generation of an induced pluripotent stem cell line (TRNDi004-I) from a Niemann-Pick disease type B patient carrying a heterozygous mutation of p. L43_A44delLA in the SMPD1 gene. Stem cell research, 37, 101436.
- Hong, J., Xu, M., Li, R., Cheng, Y. S., Kouznetsova, J., Beers, J., … & Zheng, W. (2019). Generation of an induced pluripotent stem cell line (TRNDi008-A) from a Hunter syndrome patient carrying a hemizygous 208insC mutation in the IDS gene. Stem cell research, 37, 101451.
- Cheng, Y. S., Li, R., Baskfield, A., Beers, J., Zou, J., Liu, C., & Zheng, W. (2019). A human induced pluripotent stem cell line (TRNDi007-B) from an infantile onset Pompe patient carrying p. R854X mutation in the GAA gene. Stem cell research, 37, 101435.
- Yang, S., Cheng, Y. S., Li, R., Pradhan, M., Hong, J., Beers, J., … & Zheng, W. (2019). An induced pluripotent stem cell line (TRNDi010-C) from a patient carrying a homozygous p. R401X mutation in the NGLY1 gene. Stem cell research, 39, 101496.
- Baskfield, A., Li, R., Beers, J., Zou, J., Liu, C., & Zheng, W. (2019). An induced pluripotent stem cell line (TRNDi009-C) from a Niemann-Pick disease type A patient carrying a heterozygous p. L302P (c. 905 T> C) mutation in the SMPD1 gene. Stem cell research, 38, 101461.
- Huang, W., Xu, M., Li, R., Baskfield, A., Kouznetsova, J., Beers, J., … & Zheng, W. (2019). An induced pluripotent stem cell line (TRNDi006-A) from a MPS IIIB patient carrying homozygous mutation of p. Glu153Lys in the NAGLU gene. Stem Cell Research, 101427.
- Sundararaj, K. P., Rodgers, J., Angel, P., Wolf, B., & Nowling, T. K. (2020). Neuraminidase activity mediates IL-6 production through TLR4 and p38/ERK MAPK signaling in MRL/lpr mesangial cells. bioRxiv.
- Li, R., Baskfield, A., Lin, Y., Beers, J., Zou, J., Liu, C., … & Zheng, W. (2019). Generation of an induced pluripotent stem cell line (TRNDi003-A) from a Noonan syndrome with multiple lentigines (NSML) patient carrying a p. Q510P mutation in the PTPN11 gene. Stem cell research, 34, 101374.
- Li, R., Pradhan, M., Xu, M., Baskfield, A., Farkhondeh, A., Cheng, Y. S., … & Rodems, S. (2018). Generation of an induced pluripotent stem cell line (TRNDi002-B) from a patient carrying compound heterozygous p. Q208X and p. G310G mutations in the NGLY1 gene. Stem Cell Research, 101362.
- Poli, M. C., Ebstein, F., Nicholas, S. K., de Guzman, M. M., Forbes, L. R., Chinn, I. K., … & Coban-Akdemir, Z. H. (2018). Heterozygous Truncating Variants in POMP Escape Nonsense-Mediated Decay and Cause a Unique Immune Dysregulatory Syndrome. The American Journal of Human Genetics, 102, 1-17. https://doi.org/10.1016/j.ajhg.2018.04.010
- Vozdek, R., Long, Y., & Ma, D. K. (2018). The receptor tyrosine kinase HIR-1 coordinates HIF-independent responses to hypoxia and extracellular matrix injury. Sci. Signal., 11(550), eaat0138
PRODUCTS
Genome Editing Kits for Knock-in, Knockout, CKO Mouse generation
Custom Mouse Injection-Ready Kit (Validated)
- Smalley, E. (2016) CRISPR mouse model boom, rat model renaissance. Nature Biotechnology. 34: 893-894
- Baker, M. (2014) Gene editing at CRISPR speed. Nature Biotechnology 32, 309–312.
- Shen, B., et al. (2013). Cell Research, 23(5), 720–723.
- Mali, P., et al. (2013) Science Express, 339, 823-826
- Ran, FA., et al. (2013). Nature protocols, 8(11), 2281-2308.
- Cho, SW., et al. (2013). Nature biotechnology, 31(3), 230-232.
- Cong, L., et al. (2013). Science (New York, N.Y.), 339(6121), 819–823.
In vivo validated Cas9 mRNAs
- Cong, L., et al. (2013). Science (New York, N.Y.), 339(6121), 819–823.
- Ran, FA, et al. (2013). Cell, 154(6), 1380-1389.
- Wang, H., et al. (2013). Cell, 153(4), 910–918. http://doi.org/10.1016/j.cell.2013.04.025.
Cell Line Generation: Genome Editing Products
TARGATT™ Site-Specific Knock-in Cell Lines
Book Chapters
- Chen-Tsai, R. Y. (2020). Integrase-Mediated Targeted Transgenics Through Pronuclear Microinjection. In Transgenic Mouse (pp. 35-46). Humana, New York, NY.
- Chen-Tsai, R. Y. (2019). Using TARGATT™ Technology to Generate Site-Specific Transgenic Mice. In Microinjection (pp. 71-86). Humana Press, New York, NY.
Master Cell Line
- Chi, X., Zheng, Q., Jiang, R., Chen-Tsai, R. Y., & Kong, L. J. (2019). A system for site-specific integration of transgenes in mammalian cells. PLOS ONE, 14(7), e0219842.
Description of the technology
- Zhu, F., Gamboa, M., Farruggio, A. P., Hippenmeyer, S., Tasic, B., Schüle, B., … Calos, M. P. (2014). DICE, an efficient system for iterative genomic editing in human pluripotent stem cells. Nucleic Acids Research, 42(5), e34. http://doi.org/10.1093/nar/gkt1290.
- Tasic, B., Hippenmeyer, S., Wang, C., Gamboa, M., Zong, H., Chen-Tsai, Y., & Luo, L. (2011). Site-specific integrase-mediated transgenesis in mice via pronuclear injection. Proceedings of the National Academy of Sciences of the United States of America, 108(19), 7902–7907. http://doi.org/10.1073/pnas.1019507108.
Commentary, comparison with other transgenic methods
- Rossant, J., Nutter, L. M., & Gertsenstein, M. (2011). Engineering the embryo. Proceedings of the National Academy of Sciences, 108(19), 7659-7660.
Tet inducible mice generated by TARGATT™
- Fan, X., Petitt, M., Gamboa, M., Huang, M., Dhal, S., Druzin, M. L., … Nayak, N. R. (2012). Transient, Inducible, Placenta-Specific Gene Expression in Mice. Endocrinology, 153(11), 5637–5644. http://doi.org/10.1210/en.2012-1556.
Advantage of Hipp11 (H11) locus
- Hippenmeyer, S., Youn, Y. H., Moon, H. M., Miyamichi, K., Zong, H., Wynshaw-Boris, A., & Luo, L. (2010). Genetic Mosaic Dissection of Lis1 and Ndel1 in Neuronal Migration. Neuron, 68(4), 695–709. http://doi.org/10.1016/j.neuron.2010.09.027.
Applications for mice generated by TARGATT™ (and cited/published articles)
- Carlson, H. L., & Stadler, H. S. (2019). Development and functional characterization of a lncRNA‐HIT conditional loss of function allele. genesis, e23351.
- Chande, S., Ho, B., Fetene, J., & Bergwitz, C. (2019). Transgenic mouse model for conditional expression of influenza hemagglutinin-tagged human SLC20A1/PIT1. PloS one, 14(10), e0223052. doi:10.1371/journal.pone.0223052
- Hu, Q., Ye, Y., Chan, L. C., Li, Y., Liang, K., Lin, A., … & Pan, Y. (2019). Oncogenic lncRNA downregulates cancer cell antigen presentation and intrinsic tumor suppression. Nature immunology, 1.
- Matharu, N., Rattanasopha, S., Tamura, S., Maliskova, L., Wang, Y., Bernard, A., … & Ahituv, N. (2018). CRISPR-mediated activation of a promoter or enhancer rescues obesity caused by haploinsufficiency. Science, eaau0629.
- Barrett, R. D., Laurent, S., Mallarino, R., Pfeifer, S. P., Xu, C. C., Foll, M., … & Hoekstra, H. E. (2018). The fitness consequences of genetic variation in wild populations of mice. bioRxiv, 383240.
- Ibrahim, L. A., Huang, J. J., Wang, S. Z., Kim, Y. J., Li, I., & Huizhong, W. (2018). Sparse Labeling and Neural Tracing in Brain Circuits by STARS Strategy: Revealing Morphological Development of Type II Spiral Ganglion Neurons. Cerebral Cortex, 1-14.
- Kumar, A., Dhar, S., Campanelli, G., Butt, N. A., Schallheim, J. M., Gomez, C. R., & Levenson, A. S. (2018). MTA 1 drives malignant progression and bone metastasis in prostate cancer. Molecular oncology.
- Jang, Y., Wang, C., Broun, A., Park, Y. K., Zhuang, L., Lee, J. E., … & Ge, K. (2018). H3. 3K4M destabilizes enhancer epigenomic writers MLL3/4 and impairs adipose tissue development. bioRxiv, 301986. doi:https://doi.org/10.1101/301986
- Tang, Y., Kwon, H., Neel, B. A., Kasher-Meron, M., Pessin, J., Yamada, E., & Pessin, J. E. (2018). The fructose-2, 6-bisphosphatase TIGAR suppresses NF-κB signaling by directly inhibiting the linear ubiquitin assembly complex LUBAC. Journal of Biological Chemistry, jbc-RA118.
- Chen, M., Geoffroy, C. G., Meves, J. M., Narang, A., Li, Y., Nguyen, M. T., … & Elzière, L. (2018). Leucine Zipper-Bearing Kinase Is a Critical Regulator of Astrocyte Reactivity in the Adult Mammalian CNS. Cell Reports, 22(13), 3587-3597.
- Kido, T., Sun, Z., & Lau, Y.-F. C. (2017). Aberrant activation of the human sex-determining gene in early embryonic development results in postnatal growth retardation and lethality in mice. Scientific Reports, 7, 4113. http://doi.org/10.1038/s41598-017-04117-6.
- Nouri, N., & Awatramani, R. (2017). A novel floor plate boundary defined by adjacent En1 and Dbx1 microdomains distinguishes midbrain dopamine and hypothalamic neurons. Development, 144(5), 916-927.
- Li, K., Wang, F., Cao, W. B., Lv, X. X., Hua, F., Cui, B., … & Yu, J. M. (2017). TRIB3 Promotes APL Progression through Stabilization of the Oncoprotein PML-RARα and Inhibition of p53-Mediated Senescence. Cancer Cell, 31(5), 697-710.
- Matharu, N., Rattanasopha, S., Maliskova, L., Wang, Y., Hardin, A., Vaisse, C., & Ahituv, N. (2017). Promoter or Enhancer Activation by CRISPRa Rescues Haploinsufficiency Caused Obesity. bioRxiv, 140426.
- Jiang, T., Kindt, K., & Wu, D. K. (2017). Transcription factor Emx2 controls stereociliary bundle orientation of sensory hair cells. eLife, 6, e23661.
- Booze, M. L., Hansen, J. M., & Vitiello, P. F. (2016). A Novel Mouse Model for the Identification of Thioredoxin-1 Protein Interactions. Free Radical Biology & Medicine, 99, 533–543. http://doi.org/10.1016/j.freeradbiomed.2016.09.013.
- Feng, D., Dai, S., Liu, F., Ohtake, Y., Zhou, Z., Wang, H., … & Hayat, U. (2016). Cre-inducible human CD59 mediates rapid cell ablation after intermedilysin administration. The Journal of clinical investigation, 126(6), 2321-2333.
- Sun, N., Yun, J., Liu, J., Malide, D., Liu, C., Rovira, I. I., … Finkel, T. (2015). Measuring in vivo mitophagy. Molecular Cell, 60(4), 685–696. http://doi.org/10.1016/j.molcel.2015.10.009.
- Devine, W. P., Wythe, J. D., George, M., Koshiba-Takeuchi, K., & Bruneau, B. G. (2014). Early patterning and specification of cardiac progenitors in gastrulating mesoderm. eLife, 3, e03848. http://doi.org/10.7554/eLife.03848.
- Fogg, P. C. M., Colloms, S., Rosser, S., Stark, M., & Smith, M. C. M. (2014). New Applications for Phage Integrases. Journal of Molecular Biology, 426(15), 2703–2716. http://doi.org/10.1016/j.jmb.2014.05.014.
- Chen-Tsai, R. Y., Jiang, R., Zhuang, L., Wu, J., Li, L., & Wu, J. (2014). Genome editing and animal models. Chinese science bulletin, 59(1), 1-6.
- Park, K.-E., Park, C.-H., Powell, A., Martin, J., Donovan, D. M., & Telugu, B. P. (2016). Targeted Gene Knockin in Porcine Somatic Cells Using CRISPR/Cas Ribonucleoproteins. International Journal of Molecular Sciences, 17(6), 810. http://doi.org/10.3390/ijms17060810.
- Guenther, C. A., Tasic, B., Luo, L., Bedell, M. A., & Kingsley, D. M. (2014). A molecular basis for classic blond hair color in Europeans. Nature Genetics, 46(7), 748–752. http://doi.org/10.1038/ng.2991.
- Villamizar, C. A. (2014). Characterization of the vascular pathology in the acta2 r258c mouse model and cerebrovascular characterization of the acta2 null mouse. UT GSBS Dissertations and These (Open Access). Paper 508 (2014)
CRISPR Cell Line Knock-Out, Knock-in, Point Mutation
- Tanic, J., Wang, Y., Lee, W., Coelho, N. M., Glogauer, M., & McCulloch, C. A. (2019). Adseverin modulates morphology and invasive function of MCF7 cells. Biochimica et Biophysica Acta (BBA)-Molecular Basis of Disease, 1865(10), 2716-2725.
- Panda, D., Gjinaj, E., Bachu, M., Squire, E., Novatt, H., Ozato, K., & Rabin, R. L. (2019). IRF1 maintains optimal constitutive expression of antiviral genes and regulates the early antiviral response. Frontiers in immunology, 10, 1019.
- Pisapia, P., Malapelle, U., Roma, G., Saddar, S., Zheng, Q., Pepe, F., … & Nikiforov, Y. E. (2019). Consistency and reproducibility of next‐generation sequencing in cytopathology: A second worldwide ring trial study on improved cytological molecular reference specimens. Cancer cytopathology, 127(5), 285-296.
- Ilic, D. (2019). Latest developments in the field of stem cell research and regenerative medicine compiled from publicly available information and press releases from nonacademic institutions in October 2018. Regenerative medicine, 14(2), 85-92.
- Simkin, D., Searl, T. J., Piyevsky, B. N., Forrest, M., Williams, L. A., Joshi, V., … & Penzes, P. (2019). Impaired M-current in KCNQ2 Encephalopathy Evokes Dyshomeostatic Modulation of Excitability. bioRxiv, 538371. https://doi.org/10.1101/538371
- Jang, Y., Choi, J., Park, N., Kang, J., Kim, M., Kim, Y., & Ju, J. H. (2019). Development of immunocompatible pluripotent stem cells via CRISPR-based human leukocyte antigen engineering. Experimental & Molecular Medicine, 51(1), 3.
- Colomar-Carando, N., Meseguer, A., Jutz, S., Herrera-Fernández, V., Olvera, A., Kiefer, K., … & Vicente, R. (2018). Zip6 Transporter Is an Essential Component of the Lymphocyte Activation Machinery. The Journal of Immunology, ji1800689.
- Tanic, J. (2018). A Role for Adseverin in the Invasion and Migration of MCF7 Breast Adenocarcinoma Cells (Doctoral dissertation).
- Lizarraga, S. B., Maguire, A. M., Ma, L., van Dyck, L. I., Wu, Q., Nagda, D., … & Cowen, M. H. (2018). Human neurons from Christianson syndrome iPSCs reveal allele-specific responses to rescue strategies. bioRxiv, 444232.
- Tanaka, H., Kondo, K., Chen, X., Homma, H., Tagawa, K., Kerever, A., … & Fujita, K. (2018). The intellectual disability gene PQBP1 rescues Alzheimer’s disease pathology. Molecular Psychiatry, 1.
- Yin, Y., Garcia, M. R., Novak, A. J., Saunders, A. M., Ank, R. S., Nam, A. S., & Fisher, L. W. (2018). Surf4 (Erv29p) binds amino-terminal tripeptide motifs of soluble cargo proteins with different affinities, enabling prioritization of their exit from the endoplasmic reticulum. PLoS biology, 16(8), e2005140.
- Selvan, N., George, S., Serajee, F. J., Shaw, M., Hobson, L., Kalscheuer, V. M., … & Schwartz, C. E. (2018). O-GlcNAc transferase missense mutations linked to X-linked intellectual disability deregulate genes involved in cell fate determination and signaling. Journal of Biological Chemistry, jbc-RA118.
- Smalley, E. (2018). FDA warns public of dangers of DIY gene therapy. https://doi.org/10.1038/nbt0218-119
- Chai, S., Wan, X., Ramirez-Navarro, A., Tesar, P. J., Kaufman, E. S., Ficker, E., … & Deschênes, I. (2018). Physiological genomics identifies genetic modifiers of long QT syndrome type 2 severity. The Journal of clinical investigation, 128(3).
- Boi, S., Ferrell, M. E., Zhao, M., Hasenkrug, K. J., & Evans, L. H. (2018). Mouse APOBEC3 expression in NIH 3T3 cells mediates hypermutation of AKV murine leukemia virus. Virology, 518, 377-384. https://doi.org/10.1016/j.virol.2018.03.014.
- Molinski, S. V., et al. (2017). Orkambi® and amplifier co‐therapy improves function from a rare CFTR mutation in gene‐edited cells and patient tissue. EMBO Molecular Medicine, e201607137.
- Petrovic, P. B. (2017). Myosin Phosphatase Rho-interacting Protein Regulates DDR1-mediated Collagen Tractional Remodeling (Doctoral dissertation, University of Toronto (Canada)).
- Peng, L., Zhang, H., Hao, Y., Xu, F., Yang, J., Zhang, R., … & Chen, C. (2016). Reprogramming macrophage orientation by microRNA 146b targeting transcription factor IRF5. EBioMedicine, 14, 83-96.
- Hu, J. K., Crampton, J. C., Locci, M., & Crotty, S. (2016). CRISPR-mediated Slamf1Δ/Δ Slamf5Δ/Δ Slamf6Δ/Δ triple gene disruption reveals NKT cell defects but not T follicular helper cell defects. PloS one, 11(5), e0156074.
- Smalley, E. (2016). CRISPR mouse model boom, rat model renaissance. Nature Biotechnology. 34, 893–894.
- Baker, M. (2014). Gene editing at CRISPR speed. Nature biotechnology, 32(4), 309-313.
TARGATT™ Products for Site-Specific Knock-in Mouse Generation
Book Chapters
- Chen-Tsai, R. Y. (2020). Integrase-Mediated Targeted Transgenics Through Pronuclear Microinjection. In Transgenic Mouse (pp. 35-46). Humana, New York, NY.
- Chen-Tsai, R. Y. (2019). Using TARGATT™ Technology to Generate Site-Specific Transgenic Mice. In Microinjection (pp. 71-86). Humana Press, New York, NY.
Master Cell Line
- Chi, X., Zheng, Q., Jiang, R., Chen-Tsai, R. Y., & Kong, L. J. (2019). A system for site-specific integration of transgenes in mammalian cells. PLOS ONE, 14(7), e0219842.
Description of the technology
- Zhu, F., Gamboa, M., Farruggio, A. P., Hippenmeyer, S., Tasic, B., Schüle, B., … Calos, M. P. (2014). DICE, an efficient system for iterative genomic editing in human pluripotent stem cells. Nucleic Acids Research, 42(5), e34. http://doi.org/10.1093/nar/gkt1290.
- Tasic, B., Hippenmeyer, S., Wang, C., Gamboa, M., Zong, H., Chen-Tsai, Y., & Luo, L. (2011). Site-specific integrase-mediated transgenesis in mice via pronuclear injection. Proceedings of the National Academy of Sciences of the United States of America, 108(19), 7902–7907. http://doi.org/10.1073/pnas.1019507108.
Commentary, comparison with other transgenic methods
- Rossant, J., Nutter, L. M., & Gertsenstein, M. (2011). Engineering the embryo. Proceedings of the National Academy of Sciences, 108(19), 7659-7660.
Tet inducible mice generated by TARGATT™
- Fan, X., Petitt, M., Gamboa, M., Huang, M., Dhal, S., Druzin, M. L., … Nayak, N. R. (2012). Transient, Inducible, Placenta-Specific Gene Expression in Mice. Endocrinology, 153(11), 5637–5644. http://doi.org/10.1210/en.2012-1556.
Advantage of Hipp11 (H11) locus
- Hippenmeyer, S., Youn, Y. H., Moon, H. M., Miyamichi, K., Zong, H., Wynshaw-Boris, A., & Luo, L. (2010). Genetic Mosaic Dissection of Lis1 and Ndel1 in Neuronal Migration. Neuron, 68(4), 695–709. http://doi.org/10.1016/j.neuron.2010.09.027.
Applications for mice generated by TARGATT™ (and cited/published articles)
- Lindtner, S., Catta-Preta, R., Tian, H., Su-Feher, L., Price, J. D., Dickel, D. E., … & Pennacchio, L. A. (2019). Genomic Resolution of DLX-Orchestrated Transcriptional Circuits Driving Development of Forebrain GABAergic Neurons. Cell reports, 28(8), 2048-2063.
- Wang, T. A., Teo, C. F., Åkerblom, M., Chen, C., Tynan-La Fontaine, M., Greiner, V. J., … & Jan, L. Y. (2019). Thermoregulation via Temperature-Dependent PGD2 Production in Mouse Preoptic Area. Neuron, 103(2), 309-322.
- Clarke, B. A., Majumder, S., Zhu, H., Lee, Y. T., Kono, M., Li, C., … & Byrnes, C. (2019). The Ormdl genes regulate the sphingolipid synthesis pathway to ensure proper myelination and neurologic function in mice. eLife, 8.
- Carlson, H. L., & Stadler, H. S. (2019). Development and functional characterization of a lncRNA‐HIT conditional loss of function allele. genesis, e23351.
- Chande, S., Ho, B., Fetene, J., & Bergwitz, C. (2019). Transgenic mouse model for conditional expression of influenza hemagglutinin-tagged human SLC20A1/PIT1. PloS one, 14(10), e0223052. doi:10.1371/journal.pone.0223052
- Hu, Q., Ye, Y., Chan, L. C., Li, Y., Liang, K., Lin, A., … & Pan, Y. (2019). Oncogenic lncRNA downregulates cancer cell antigen presentation and intrinsic tumor suppression. Nature immunology, 1.
- Matharu, N., Rattanasopha, S., Tamura, S., Maliskova, L., Wang, Y., Bernard, A., … & Ahituv, N. (2018). CRISPR-mediated activation of a promoter or enhancer rescues obesity caused by haploinsufficiency. Science, eaau0629.
- Barrett, R. D., Laurent, S., Mallarino, R., Pfeifer, S. P., Xu, C. C., Foll, M., … & Hoekstra, H. E. (2018). The fitness consequences of genetic variation in wild populations of mice. bioRxiv, 383240.
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Genome Editing Kits for Knock-in, Knockout, CKO Rat generation
CRISPRCRITTERS™ Custom Rat Injection-Ready Kit (Validated)
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Patient Samples, Cell Pellets, Immortalized Fibroblasts
Human Primary Cell (Normal)
ASE-5168
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ASE-5063 – Human Kidney Epithelial Cells (Healthy)
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Human Skin Fibroblast Total RNA
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Total RNA-ASD-0088
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Human Cardiomyocytes Total RNA-ASD-0061
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HEK293T cells
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Murine Endothelial cells
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FFPE Reference Standards
MAPK Mutation Panel FFPE Reference Standards
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Cell Culture: MEF Feeder Cells, Stem Cell Grade FBS
MEF Feeder Cells
DR4 MEF Feeder Cells
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CF-1 MEF Feeder Cells
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Neo-resistant MEF Feeder Cells
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SNL 76/7 (STO Cell Line)
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ESC-Sure™ FBS
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- Marian, C. A., Stoszko, M., Wang, L., Leighty, M. W., de Crignis, E., Maschinot, C. A., … & Duvall, J. R. (2018). Small Molecule Targeting of Specific BAF (mSWI/SNF) Complexes for HIV Latency Reversal. Cell Chemical Biology.
- Hodges, H. C., Stanton, B. Z., Cermakova, K., Chang, C. Y., Miller, E. L., Kirkland, J. G., … & Crabtree, G. R. (2017). Dominant-negative SMARCA4 mutants alter the accessibility landscape of tissue-unrestricted enhancers. Nature Structural & Molecular Biology, 1.
- Braun, S. M. G., Kirkland, J. G., Chory, E. J., Husmann, D., Calarco, J. P., & Crabtree, G. R. (2017). Rapid and reversible epigenome editing by endogenous chromatin regulators. Nature Communications, 8, 560.http://doi.org/10.1038/s41467-017-00644-y.s
- Dykhuizen, E. C., Carmody, L. C., & Tolliday, N. J. (2017). High-Throughput Screening of Small Molecule Transcriptional Regulators in Embryonic Stem Cells Using qRT-PCR. In Epigenetics and Gene Expression in Cancer, Inflammatory and Immune Diseases (pp. 81-95). Humana Press, New York, NY.
- Stanton, B. Z., Hodges, C., Calarco, J. P., Braun, S. M. G., Ku, W. L., Kadoch, C., … Crabtree, G. R. (2017). SMARCA4 ATPase mutations disrupt direct eviction of PRC1 from chromatin. Nature Genetics, 49(2), 282–288. http://doi.org/10.1038/ng.3735
- Beske, P. H., Bradford, A. B., Grynovicki, J. O., Glotfelty, E. J., Hoffman, K. M., Hubbard, K. S., … & McNutt, P. M. (2015). Botulinum and tetanus neurotoxin-induced blockade of synaptic transmission in networked cultures of human and rodent neurons. Toxicological Sciences, 149(2), 503-515. doi: 10.1093/toxsci/kfv254
- Miljan, E. (2015) The Business of Stem Cell Research Tools, in Stem Cells in Regenerative Medicine: Science, regulation and business strategies (eds A. A. Vertès, N. Qureshi, A. I. Caplan and L. E. Babiss), John Wiley & Sons, Ltd, Chichester, UK. doi: 10.1002/9781118846193.ch8.
- Hubbard, K., Beske, P., Lyman, M., & McNutt, P. (2015). Functional evaluation of biological neurotoxins in networked cultures of stem cell-derived central nervous system neurons. Journal of visualized experiments: JoVE, (96).
- Stanford Medicine Transgenic Research center (http://med.stanford.edu/transgenic/esmeflif.html)
- Hathaway, N. A., Bell, O., Hodges, C., Miller, E. L., Neel, D. S., & Crabtree, G. R. (2012). Dynamics and memory of heterochromatin in living cells. Cell, 149(7), 1447-1460.
Stem Cell Research
iPSC/ESCs
Control iPSC Lines:
- Tanaka, H., Homma, H., Fujita, K., Kondo, K., Yamada, S., Jin, X., … & Atsuta, N. (2020). YAP-dependent necrosis occurs in early stages of Alzheimer’s disease and regulates mouse model pathology. Nature Communications, 11(1), 1-22.
- Su, S., Guntur, A. R., Nguyen, D. C., Fakory, S. S., Doucette, C. C., Leech, C., … & Sims-Lucas, S. (2018). A renewable source of human beige adipocytes for development of therapies to treat metabolic syndrome. Cell reports, 25(11), 3215-3228.
- Lizarraga, S. B., Maguire, A. M., Ma, L., van Dyck, L. I., Wu, Q., Nagda, D., … & Cowen, M. H. (2018). Human neurons from Christianson syndrome iPSCs reveal allele-specific responses to rescue strategies. bioRxiv, 444232.
- Tanaka, H., Kondo, K., Chen, X., Homma, H., Tagawa, K., Kerever, A., … & Fujita, K. (2018). The intellectual disability gene PQBP1 rescues Alzheimer’s disease pathology. Molecular Psychiatry, 1.
- Kavyasudha C., Macrin D., ArulJothi K.N., Joseph J.P., Harishankar M.K., Devi A. (2018) Clinical Applications of Induced Pluripotent Stem Cells – Stato Attuale. In: Advances in Experimental Medicine and Biology. Springer, New York, NY. https://doi.org/10.1007/5584_2018_173.
- Lin, Y., Linask, K. L., Mallon, B., Johnson, K., Klein, M., Beers, J., … & Zou, J. (2017). Heparin Promotes Cardiac Differentiation of Human Pluripotent Stem Cells in Chemically Defined Albumin‐Free Medium, Enabling Consistent Manufacture of Cardiomyocytes. Stem cells translational medicine, 6(2), 527-538.
ASE-9208: Sporadic Parkinson’s disease line
- Hsieh, C. H., Shaltouki, A., Gonzalez, A. E., da Cruz, A. B., Burbulla, L. F., Lawrence, E. S., … & Wang, X. (2016). Functional impairment in Miro degradation and mitophagy is a shared feature in familial and sporadic Parkinson’s disease. Cell Stem Cell, 19(6), 709-724.
iPSC-differentiated cell lines
- Gupta, G., Gliga, A., Hedberg, J., Serra, A., Greco, D., Odnevall Wallinder, I., & Fadeel, B. Cobalt nanoparticles trigger ferroptosis‐like cell death (oxytosis) in neuronal cells: Potential implications for neurodegenerative disease. The FASEB Journal.
- Kussauer, S., David, R., & Lemcke, H. (2019). hiPSCs Derived Cardiac Cells for Drug and Toxicity Screening and Disease Modeling: What Micro-Electrode-Array Analyses Can Tell Us. Cells, 8(11), 1331.
- Cheng, F., Fransson, L. Å., & Mani, K. (2019). The cyanobacterial neurotoxin β-N-methylamino-l-alanine prevents addition of heparan sulfate to glypican-1 and increases processing of amyloid precursor protein in dividing neuronal cells. Experimental Cell Research. https://doi.org/10.1016/j.yexcr.2019.03.041
- Daily, N. J., et al. (2017). High-Throughput Phenotyping of Human Induced Pluripotent Stem Cell-Derived Cardiomyocytes and Neurons Using Electric Field Stimulation and High-Speed Fluorescence Imaging. ASSAY and Drug Development Technologies. 15(4): 178-188. https://doi.org/10.1089/adt.2017.781
- Daily, N. J., Santos, R., Vecchi, J., Kemanli, P., & Wakatsuki, T. (2017). Calcium transient assays for compound screening with human iPSC-derived cardiomyocytes: Evaluating new tools. Journal of evolving stem cell research, 1(2), 1.
- Daily, N. J., et al. (2015). Journal of Bioengineering & Biomedical Science, 2015.
Mouse/ Rat/ Pig/ Guinea Pig
- ASE-9005: GermLine mESC C57Bl6/129SvJ
- Lee, S. B., et al. (2012). Stem Cells, 30(5), 997-1007.
- Imai K (2012) Applied Surface Science 258:8444-8447 (P.8445).
- Imai K (2011) J Oral Tissue Engin 8: 203-211.
- Besschetnova, TY., et al. (2015). Matrix Biology, 42, 56-73
3D-iPSC Matrix
- Li, L., & LaBarbera, D. V. (2017). 3D High-Content Screening of Organoids for Drug Discovery. Reference Module in Chemistry, Molecular Sciences and Chemical Engineering. 388-415. doi.org/10.1016/B978-0-12-409547-2.12329-7
Stem Cell Characterization
Anti-SOX2 antibody:
- Wang, T., Choi, E., Monaco, M. C. G., Major, E. O., Medynets, M., & Nath, A. (2015). Direct Induction of Human Neural Stem Cells from Peripheral Blood Hematopoietic Progenitor Cells. Journal of Visualized Experiments : JoVE, (95), 52298. Advance online publication. http://doi.org/10.3791/52298.
- Almuraikhi, N. (2015). Direct differentiation of human iPS cells towards the erythroid lineage. Ph.D. Dissertation.
Anti-TRA-1-60 antibody:
- Ramalingam, S., London, V., Kandavelou, K., Cebotaru, L., Guggino, W., Civin, C., & Chandrasegaran, S. (2013). Generation and Genetic Engineering of Human Induced Pluripotent Stem Cells Using Designed Zinc Finger Nucleases. Stem Cells and Development, 22(4), 595–610. http://doi.org/10.1089/scd.2012.0245.
Mouse ES-iPSC Characterization kit:
- Grabundzija, I., Wang, J., Sebe, A., Erdei, Z., Kajdi, R., Devaraj, A., … Ivics, Z. (2013). Sleeping Beauty transposon-based system for cellular reprogramming and targeted gene insertion in induced pluripotent stem cells. Nucleic Acids Research, 41(3), 1829–1847. http://doi.org/10.1093/nar/gks1305.
- Tsai, Y. T. (2018). Therapy and mechanism of Mendelian eye diseases (Doctoral dissertation, Columbia University).
- Yang, J., Li, Y., Erol, D., Wu, W.-H., Tsai, Y.-T., Li, X.-R., … Tsang, S. H. (2014). Generation of Induced Pluripotent Stem Cells from Conjunctiva. Graefe’s Archive for Clinical and Experimental Ophthalmology = Albrecht von Graefes Archiv Fur Klinische Und Experimentelle Ophthalmologie, 252(3), 423–431. http://doi.org/10.1007/s00417-014-2575-9.
Human ES-iPSC Characterization kit:
- Southard, S. M., Kotipatruni, R. P., & Rust, W. L. (2018). Generation and selection of pluripotent stem cells for robust differentiation to insulin-secreting cells capable of reversing diabetes in rodents. PloS one, 13(9), e0203126
- Tang, R., Jing, L., Willard, V. P., Wu, C. L., Guilak, F., Chen, J., & Setton, L. A. (2018). Differentiation of human induced pluripotent stem cells into nucleus pulposus-like cells. Stem Cell Research & Therapy, 9(1), 61.
- Marei, H. E., Althani, A., Lashen, S., Cenciarelli, C., & Hasan, A. (2017). Genetically unmatched human iPSC and ESC exhibit equivalent gene expression and neuronal differentiation potential. Scientific Reports, 7(1), 17504.
- Choi, J., Lee, S., Clement, K., Mallard, W., Tagliazucchi, G. M., Lim, H., … Hochedlinger, K. (2015). A comparison of genetically matched cell lines reveals the equivalence of human iPSCs and ESCs. Nature Biotechnology, 33(11), 1173–1181. http://doi.org/10.1038/nbt.3388
- Martí Gutiérrez, N. (2015). Doctoral Thesis, Departamento de Bioquímica y Biología Molecular, Universidad de Valencia.
- Fleming, W. H., & Hirschman, S. Z. (2015). U.S. Patent Application No. 14/410,508.
- Wang, D. (2015). Individualized Cardiovascular Medicine: Identifying New Mechanisms to Inhibit the Development of Myointimal Hyperplasia.
- Cassidy, L., Diaz, R., Chen-Tsai, R. Y., & Seigel, G. M. (2014). Comparative analysis of ABCG2+ and ABCG2-retinoblastoma cells in three-dimensional culture. Edorium Journal of Otolaryngology, 4, 1-7.
- Li, Y., Wu, W.-H., Hsu, C.-W., Nguyen, H. V., Tsai, Y.-T., Chan, L., … Tsang, S. H. (2014). Gene Therapy in Patient-specific Stem Cell Lines and a Preclinical Model of Retinitis Pigmentosa With Membrane Frizzled-related Protein Defects. Molecular Therapy, 22(9), 1688–1697. http://doi.org/10.1038/mt.2014.100
- Yagyu, S., Hoyos, V., Del Bufalo, F., & Brenner, M. K. (2015). An inducible caspase-9 suicide gene to improve the safety of therapy using human induced pluripotent stem cells. Molecular Therapy, 23(9), 1475-1485.
- Li, Y., Tsai, Y.-T., Hsu, C.-W., Erol, D., Yang, J., Wu, W.-H., … Tsang, S. H. (2012). Long-term Safety and Efficacy of Human-Induced Pluripotent Stem Cell (iPS) Grafts in a Preclinical Model of Retinitis Pigmentosa. Molecular Medicine, 18(1), 1312–1319. http://doi.org/10.2119/molmed.2012.00242
- Jing, L., Christoforou, N., Leong, K. W., Setton, L. A., & Chen, J. (2012). Differentiation potential of human induced pluripotent stem cells (iPSCs) to nucleus pulposus-like cells in vitro. Global Spine Journal, 2(1_suppl), s-0032.
- Deuse, T., Seifert, M., Phillips, N., Fire, A., Tyan, D., Kay, M., … & Volk, H. D. (2011). Immunobiology of naive and genetically modified HLA-class-I-knockdown human embryonic stem cells. J Cell Sci, 124(17), 3029-3037.
- Deuse, T., Hua, X., Wang, D., Maegdefessel, L., Heeren, J., Scheja, L., … Schrepfer, S. (2014). Dichloroacetate prevents restenosis in preclinical animal models of vessel injury. Nature, 509(7502), 641–644. http://doi.org/10.1038/nature13232.
- Wang, J., Xie, G., Singh, M., Ghanbarian, A. T., Raskó, T., Szvetnik, A., … & Schumann, G. G. (2014). Primate-specific endogenous retrovirus-driven transcription defines naive-like stem cells. Nature, 516(7531), 405.
- Wang, T., Choi, E., Monaco, M. C. G., Campanac, E., Medynets, M., Do, T., … Nath, A. (2013). Derivation of Neural Stem Cells from Human Adult Peripheral CD34+ Cells for an Autologous Model of Neuroinflammation. PLoS ONE, 8(11), e81720. http://doi.org/10.1371/journal.pone.0081720.
- Zamule, S. M., Coslo, D. M., Chen, F., & Omiecinski, C. J. (2011). Differentiation of Human Embryonic Stem Cells along a Hepatic Lineage. Chemico-Biological Interactions, 190(1), 62–72. http://doi.org/10.1016/j.cbi.2011.01.009
- Sun, N., & Zhao, H. (2014). Seamless correction of the sickle cell disease mutation of the HBB gene in human induced pluripotent stem cells using TALENs. Biotechnology and bioengineering, 111(5), 1048-1053.
- Tachibana, M., Amato, P., Sparman, M., Gutierrez, N. M., Tippner-Hedges, R., Ma, H., … Mitalipov, S. (2013). Human Embryonic Stem Cells Derived by Somatic Cell Nuclear Transfer. Cell, 153(6), 1228–1238. http://doi.org/10.1016/j.cell.2013.05.006.