Gene Editing

Displaying 1 - 21 of 21CSV
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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Witte, I. P., Lampe, G. D., Eitzinger, S., Miller, S. M., Berríos, K. N., McElroy, A. N., King, R. T., Stringham, O. G., Gelsinger, D. R., Vo, P. L. H., Chen, A. T., Tolar, J., Osborn, M. J., Sternberg, S. H., & Liu, D. R. (2025). Programmable gene insertion in human cells with a laboratory-evolved CRISPR-associated transposase. Science, 388(6748). https://doi.org/10.1126/science.adt5199
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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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da Costa, B. L., Caruso, S. M., Tsai, Y.-T., Castillejos, D. S., Sylla, M., Tsang, S. H., & Quinn, P. M. J. (2025). Prime Editing Strategy to Install the RPE65 c.1430A>G Dominant Mutation. Retinal Degenerative Diseases XX, 101–106. https://doi.org/10.1007/978-3-031-76550-6_17
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da Costa, B. L., Pincay, J., Brodie, S. E., Tsang, S. H., & Quinn, P. M. J. (2025). Prime Editing Strategy to Install the Mfrp Retinal Degeneration 6 Mutation. Retinal Degenerative Diseases XX, 113–118. https://doi.org/10.1007/978-3-031-76550-6_19
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Zhu, Y., Cai, S. S., Ma, J., Cheng, L., Wei, C., Aggarwal, A., Toh, W. H., Shin, C., Shen, R., Kong, J., Mao, S. A., Lao, Y.-H., Leong, K. W., & Mao, H.-Q. (2024). Optimization of lipid nanoparticles for gene editing of the liver via intraduodenal delivery. Biomaterials, 308, 122559. https://doi.org/10.1016/j.biomaterials.2024.122559
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Szekely, O., Rangadurai, A. K., Gu, S., Manghrani, A., Guseva, S., & Al-Hashimi, H. M. (2024). NMR measurements of transient low-populated tautomeric and anionic Watson–Crick-like G·T/U in RNA:DNA hybrids: implications for the fidelity of transcription and CRISPR/Cas9 gene editing. Nucleic Acids Research, 52(5), 2672–2685. https://doi.org/10.1093/nar/gkae027
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Papaioannou, V. E., & Behringer, R. R. (2023). Recovering a Targeted Mutation in Mice from Embryonic Stem Cell Chimeras or CRISPR–Cas Founders. Cold Spring Harbor Protocols, 2024(1), pdb.over107959. https://doi.org/10.1101/pdb.over107959
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Dubey, S., Chen, Z., Jiang, Y. J., Talis, A., Molotkov, A., Ali, A., Mintz, A., & Momen-Heravi, F. (2024). Small extracellular vesicles (sEVs)-based gene delivery platform for cell-specific CRISPR/Cas9 genome editing. Theranostics, 14(7), 2777–2793. https://doi.org/10.7150/thno.92133
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George, J. T., Acree, C., Park, J.-U., Kong, M., Wiegand, T., Pignot, Y. L., Kellogg, E. H., Greene, E. C., & Sternberg, S. H. (2023). Mechanism of target site selection by type V-K CRISPR-associated transposases. Science, 382(6672). https://doi.org/10.1126/science.adj8543
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Girish, V., Lakhani, A. A., Thompson, S. L., Scaduto, C. M., Brown, L. M., Hagenson, R. A., Sausville, E. L., Mendelson, B. E., Kandikuppa, P. K., Lukow, D. A., Yuan, M. L., Stevens, E. C., Lee, S. N., Schukken, K. M., Akalu, S. M., Vasudevan, A., Zou, C., Salovska, B., Li, W., … Sheltzer, J. M. (2023). Oncogene-like addiction to aneuploidy in human cancers. Science, 381(6660). https://doi.org/10.1126/science.adg4521
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Lao, Y.-H., Ji, R., Zhou, J. K., Snow, K. J., Kwon, N., Saville, E., He, S., Chauhan, S., Chi, C.-W., Datta, M. S., Zhang, H., Quek, C. H., Cai, S. S., Li, M., Gaitan, Y., Bechtel, L., Wu, S.-Y., Lutz, C. M., Tomer, R., … Leong, K. W. (2023). Focused ultrasound–mediated brain genome editing. Proceedings of the National Academy of Sciences, 120(34). https://doi.org/10.1073/pnas.2302910120
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Vaitsiankova, A., Thakar, T., & Ciccia, A. (2023). Base-editing screens illuminate variant effects in human hematopoiesis. Cell Reports Methods, 3(7), 100541. https://doi.org/10.1016/j.crmeth.2023.100541
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Ling, J., Jenny, L. A., Zhou, A., & Tsang, S. H. (2022). Therapeutic Gene Editing in Inherited Retinal Disorders. Cold Spring Harbor Perspectives in Medicine, 13(4), a041292. https://doi.org/10.1101/cshperspect.a041292
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Schimmel, J., Muñoz-Subirana, N., Kool, H., van Schendel, R., van der Vlies, S., Kamp, J. A., de Vrij, F. M. S., Kushner, S. A., Smith, G. C. M., Boulton, S. J., & Tijsterman, M. (2023). Modulating mutational outcomes and improving precise gene editing at CRISPR-Cas9-induced breaks by chemical inhibition of end-joining pathways. Cell Reports, 42(2), 112019. https://doi.org/10.1016/j.celrep.2023.112019
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Patel, A., Iannello, G., Diaz, A. G., Sirabella, D., Thaker, V., & Corneo, B. (2022). Efficient Cas9‐based Genome Editing Using CRISPR Analysis Webtools in Severe Early‐onset‐obesity Patient‐derived iPSCs. Current Protocols, 2(8). Portico. https://doi.org/10.1002/cpz1.519
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Eisenson, D. L., Hisadome, Y., & Yamada, K. (2022). Progress in Xenotransplantation: Immunologic Barriers, Advances in Gene Editing, and Successful Tolerance Induction Strategies in Pig-To-Primate Transplantation. Frontiers in Immunology, 13. https://doi.org/10.3389/fimmu.2022.899657
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Wang, N.-K., Liu, P.-K., Kong, Y., Levi, S. R., Huang, W.-C., Hsu, C.-W., Wang, H.-H., Chen, N., Tseng, Y.-J., Quinn, P. M. J., Tai, M.-H., Lin, C.-S., & Tsang, S. H. (2021). Mouse Models of Achromatopsia in Addressing Temporal “Point of No Return” in Gene-Therapy. International Journal of Molecular Sciences, 22(15), 8069. https://doi.org/10.3390/ijms22158069
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