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Transgenic mice for in vivo epigenome editing with CRISPR-based systems.
Gemberling MP, Siklenka K, Rodriguez E, Tonn-Eisinger KR, Barrera A, Liu F, Kantor A, Li L, Cigliola V, Hazlett MF, Williams CA, Bartelt LC, Madigan VJ, Bodle JC, Daniels H, Rouse DC, Hilton IB, Asokan A, Ciofani M, Poss KD, Reddy TE, West AE, Gersbach CA. Gemberling MP, et al. Nat Methods. 2021 Aug;18(8):965-974. doi: 10.1038/s41592-021-01207-2. Epub 2021 Aug 2. Nat Methods. 2021. PMID: 34341582 Free PMC article.
An enhancer-based gene-therapy strategy for spatiotemporal control of cargoes during tissue repair.
Yan R, Cigliola V, Oonk KA, Petrover Z, DeLuca S, Wolfson DW, Vekstein A, Mendiola MA, Devlin G, Bishawi M, Gemberling MP, Sinha T, Sargent MA, York AJ, Shakked A, DeBenedittis P, Wendell DC, Ou J, Kang J, Goldman JA, Baht GS, Karra R, Williams AR, Bowles DE, Asokan A, Tzahor E, Gersbach CA, Molkentin JD, Bursac N, Black BL, Poss KD. Yan R, et al. Cell Stem Cell. 2023 Jan 5;30(1):96-111.e6. doi: 10.1016/j.stem.2022.11.012. Epub 2022 Dec 13. Cell Stem Cell. 2023. PMID: 36516837 Free PMC article.
Epigenome editing technologies for discovery and medicine.
McCutcheon SR, Rohm D, Iglesias N, Gersbach CA. McCutcheon SR, et al. Nat Biotechnol. 2024 Aug;42(8):1199-1217. doi: 10.1038/s41587-024-02320-1. Epub 2024 Jul 29. Nat Biotechnol. 2024. PMID: 39075148 Review.
The once and future gene therapy.
Bulaklak K, Gersbach CA. Bulaklak K, et al. Nat Commun. 2020 Nov 16;11(1):5820. doi: 10.1038/s41467-020-19505-2. Nat Commun. 2020. PMID: 33199717 Free PMC article.
Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models.
Hakim CH, Kumar SRP, Pérez-López DO, Wasala NB, Zhang D, Yue Y, Teixeira J, Pan X, Zhang K, Million ED, Nelson CE, Metzger S, Han J, Louderman JA, Schmidt F, Feng F, Grimm D, Smith BF, Yao G, Yang NN, Gersbach CA, Chen SJ, Herzog RW, Duan D. Hakim CH, et al. Nat Commun. 2021 Nov 24;12(1):6769. doi: 10.1038/s41467-021-26830-7. Nat Commun. 2021. PMID: 34819506 Free PMC article.
Nonviral In Vivo Delivery of CRISPR-Cas9 Using Protein-Agnostic, High-Loading Porous Silicon and Polymer Nanoparticles.
Fletcher RB, Stokes LD, Kelly IB 3rd, Henderson KM, Vallecillo-Viejo IC, Colazo JM, Wong BV, Yu F, d'Arcy R, Struthers MN, Evans BC, Ayers J, Castanon M, Weirich MJ, Reilly SK, Patel SS, Ivanova YI, Silvera Batista CA, Weiss SM, Gersbach CA, Brunger JM, Duvall CL. Fletcher RB, et al. ACS Nano. 2023 Sep 12;17(17):16412-16431. doi: 10.1021/acsnano.2c12261. Epub 2023 Aug 15. ACS Nano. 2023. PMID: 37582231 Free PMC article.
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