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Correction of recessive dystrophic epidermolysis bullosa by homology-directed repair-mediated genome editing.
Bonafont J, Mencía A, Chacón-Solano E, Srifa W, Vaidyanathan S, Romano R, Garcia M, Hervás-Salcedo R, Ugalde L, Duarte B, Porteus MH, Del Rio M, Larcher F, Murillas R. Bonafont J, et al. Among authors: vaidyanathan s. Mol Ther. 2021 Jun 2;29(6):2008-2018. doi: 10.1016/j.ymthe.2021.02.019. Epub 2021 Feb 18. Mol Ther. 2021. PMID: 33609734 Free PMC article.
Global Transcriptional Response to CRISPR/Cas9-AAV6-Based Genome Editing in CD34+ Hematopoietic Stem and Progenitor Cells.
Cromer MK, Vaidyanathan S, Ryan DE, Curry B, Lucas AB, Camarena J, Kaushik M, Hay SR, Martin RM, Steinfeld I, Bak RO, Dever DP, Hendel A, Bruhn L, Porteus MH. Cromer MK, et al. Among authors: vaidyanathan s. Mol Ther. 2018 Oct 3;26(10):2431-2442. doi: 10.1016/j.ymthe.2018.06.002. Epub 2018 Jul 11. Mol Ther. 2018. PMID: 30005866 Free PMC article.
Highly Efficient and Marker-free Genome Editing of Human Pluripotent Stem Cells by CRISPR-Cas9 RNP and AAV6 Donor-Mediated Homologous Recombination.
Martin RM, Ikeda K, Cromer MK, Uchida N, Nishimura T, Romano R, Tong AJ, Lemgart VT, Camarena J, Pavel-Dinu M, Sindhu C, Wiebking V, Vaidyanathan S, Dever DP, Bak RO, Laustsen A, Lesch BJ, Jakobsen MR, Sebastiano V, Nakauchi H, Porteus MH. Martin RM, et al. Among authors: vaidyanathan s. Cell Stem Cell. 2019 May 2;24(5):821-828.e5. doi: 10.1016/j.stem.2019.04.001. Cell Stem Cell. 2019. PMID: 31051134 Free article.
High-Efficiency, Selection-free Gene Repair in Airway Stem Cells from Cystic Fibrosis Patients Rescues CFTR Function in Differentiated Epithelia.
Vaidyanathan S, Salahudeen AA, Sellers ZM, Bravo DT, Choi SS, Batish A, Le W, Baik R, de la O S, Kaushik MP, Galper N, Lee CM, Teran CA, Yoo JH, Bao G, Chang EH, Patel ZM, Hwang PH, Wine JJ, Milla CE, Desai TJ, Nayak JV, Kuo CJ, Porteus MH. Vaidyanathan S, et al. Cell Stem Cell. 2020 Feb 6;26(2):161-171.e4. doi: 10.1016/j.stem.2019.11.002. Epub 2019 Dec 12. Cell Stem Cell. 2020. PMID: 31839569 Free PMC article.
Targeted replacement of full-length CFTR in human airway stem cells by CRISPR-Cas9 for pan-mutation correction in the endogenous locus.
Vaidyanathan S, Baik R, Chen L, Bravo DT, Suarez CJ, Abazari SM, Salahudeen AA, Dudek AM, Teran CA, Davis TH, Lee CM, Bao G, Randell SH, Artandi SE, Wine JJ, Kuo CJ, Desai TJ, Nayak JV, Sellers ZM, Porteus MH. Vaidyanathan S, et al. Mol Ther. 2022 Jan 5;30(1):223-237. doi: 10.1016/j.ymthe.2021.03.023. Epub 2021 Mar 29. Mol Ther. 2022. PMID: 33794364 Free PMC article.
High-efficiency transgene integration by homology-directed repair in human primary cells using DNA-PKcs inhibition.
Selvaraj S, Feist WN, Viel S, Vaidyanathan S, Dudek AM, Gastou M, Rockwood SJ, Ekman FK, Oseghale AR, Xu L, Pavel-Dinu M, Luna SE, Cromer MK, Sayana R, Gomez-Ospina N, Porteus MH. Selvaraj S, et al. Among authors: vaidyanathan s. Nat Biotechnol. 2024 May;42(5):731-744. doi: 10.1038/s41587-023-01888-4. Epub 2023 Aug 3. Nat Biotechnol. 2024. PMID: 37537500
533 results