Genetic Therapy
Borot, F., Humbert, O., Ehmsen, J. T., Fields, E., Kohli, S., Radtke, S., Swing, K., Pande, D., Enstrom, M. R., Laszlo, G. S., Mayuranathan, T., Ali, A. M., Weiss, M. J., Yen, J. S., Newby, G. A., Walter, R. B., Liu, D. R., Mukherjee, S., & Kiem, H.-P. (2025). Multiplex base editing to protect from CD33 directed drugs for immune and gene therapy. Nature Communications, 16(1). https://doi.org/10.1038/s41467-025-59713-2
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Godbout, K., Dugas, M., Reiken, S. R., Ramezani, S., Falle, A., Rousseau, J., Wronska, A. E., Lamothe, G., Canet, G., Lu, Y., Planel, E., Marks, A. R., & Tremblay, J. P. (2025). Universal Prime Editing Therapeutic Strategy for RyR1-Related Myopathies: A Protective Mutation Rescues Leaky RyR1 Channel. International Journal of Molecular Sciences, 26(7), 2835. https://doi.org/10.3390/ijms26072835
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Maniatis, T. (2024). From bacterial operons to gene therapy: 50 years of the journal Cell. Cell, 187(23), 6417–6420. https://doi.org/10.1016/j.cell.2024.10.037
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Zuccaro, M. V., LeDuc, C. A., & Thaker, V. V. (2024). Updates on Rare Genetic Variants, Genetic Testing, and Gene Therapy in Individuals With Obesity. Current Obesity Reports, 13(3), 626–641. https://doi.org/10.1007/s13679-024-00567-y
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Li, Y., Yang, R. R., Li, Y. S., Hsu, C. W., Jenny, L. A., Kong, Y., Ruan, M. Z., Sparrow, J. R., & Tsang, S. H. (2024). Evaluating precision medicine approaches for gene therapy in patient-specific cellular models of Bietti crystalline dystrophy. JCI insight, 9(16), e177231. https://doi.org/10.1172/jci.insight.177231
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Abela, L., Gianfrancesco, L., Tagliatti, E., Rossignoli, G., Barwick, K., Zourray, C., Reid, K. M., Budinger, D., Ng, J., Counsell, J., Simpson, A., Pearson, T. S., Edvardson, S., Elpeleg, O., Brodsky, F. M., Lignani, G., Barral, S., & Kurian, M. A. (2024). Neurodevelopmental and synaptic defects in DNAJC6 parkinsonism, amenable to gene therapy. Brain, 147(6), 2023–2037. https://doi.org/10.1093/brain/awae020
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Breazzano, M. P., Batson, S. A., Tsang, S. H., & Chen, R. W. S. (2022). Surgical Approach with Pars Plana Vitrectomy for Subretinal Gene Therapy. Retinitis Pigmentosa, 393–399. https://doi.org/10.1007/978-1-0716-2651-1_35
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Wu, W.-H., Tso, A., Breazzano, M. P., Jenny, L. A., Levi, S. R., Tsang, S. H., & Quinn, P. M. J. (2022). Culture of Human Retinal Explants for Ex Vivo Assessment of AAV Gene Delivery. Retinitis Pigmentosa, 303–311. https://doi.org/10.1007/978-1-0716-2651-1_28
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Pereira, E. M. (2022). Gene Therapy Update. Pediatrics In Review, 43(9), 536–537. https://doi.org/10.1542/pir.2021-005091
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Kajtna, J., Tsang, S. H., & Koch, S. F. (2022). Late-stage rescue of visually guided behavior in the context of a significantly remodeled retinitis pigmentosa mouse model. Cellular and Molecular Life Sciences, 79(3). https://doi.org/10.1007/s00018-022-04161-0
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Kanter, J., Walters, M. C., Krishnamurti, L., Mapara, M. Y., Kwiatkowski, J. L., Rifkin-Zenenberg, S., Aygun, B., Kasow, K. A., Pierciey, F. J., Bonner, M., Miller, A., Zhang, X., Lynch, J., Kim, D., Ribeil, J.-A., Asmal, M., Goyal, S., Thompson, A. A., & Tisdale, J. F. (2022). Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell Disease. New England Journal of Medicine, 386(7), 617–628. https://doi.org/10.1056/nejmoa2117175
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Thomsen, G., Burghes, A. H. M., Hsieh, C., Do, J., Chu, B. T. T., Perry, S., Barkho, B., Kaufmann, P., Sproule, D. M., Feltner, D. E., Chung, W. K., McGovern, V. L., Hevner, R. F., Conces, M., Pierson, C. R., Scoto, M., Muntoni, F., Mendell, J. R., & Foust, K. D. (2021). Biodistribution of onasemnogene abeparvovec DNA, mRNA and SMN protein in human tissue. Nature Medicine, 27(10), 1701–1711. https://doi.org/10.1038/s41591-021-01483-7
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Lopez‐Gomez, C., Sanchez‐Quintero, M. J., Lee, E. J., Kleiner, G., Tadesse, S., Xie, J., Akman, H. O., Gao, G., & Hirano, M. (2021). Synergistic Deoxynucleoside and Gene Therapies for Thymidine Kinase 2 Deficiency. Annals of Neurology, 90(4), 640–652. Portico. https://doi.org/10.1002/ana.26185
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