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Delivering AAV to the Central Nervous and Sensory Systems.
Peters CW, Maguire CA, Hanlon KS. Peters CW, et al. Trends Pharmacol Sci. 2021 Jun;42(6):461-474. doi: 10.1016/j.tips.2021.03.004. Epub 2021 Apr 13. Trends Pharmacol Sci. 2021. PMID: 33863599 Free PMC article. Review.
Gene therapy for tuberous sclerosis complex type 2 in a mouse model by delivery of AAV9 encoding a condensed form of tuberin.
Cheah PS, Prabhakar S, Yellen D, Beauchamp RL, Zhang X, Kasamatsu S, Bronson RT, Thiele EA, Kwiatkowski DJ, Stemmer-Rachamimov A, György B, Ling KH, Kaneki M, Tannous BA, Ramesh V, Maguire CA, Breakefield XO. Cheah PS, et al. Sci Adv. 2021 Jan 8;7(2):eabb1703. doi: 10.1126/sciadv.abb1703. Print 2021 Jan. Sci Adv. 2021. PMID: 33523984 Free PMC article.
In vivo Treatment of a Severe Vascular Disease via a Bespoke CRISPR-Cas9 Base Editor.
Alves CRR, Das S, Krishnan V, Ha LL, Fox LR, Stutzman HE, Shamber CE, Kalailingam P, McCarthy S, Lino Cardenas CL, Fong CE, Imai T, Mitra S, Yun S, Wood RK, Benning FMC, Lawton J, Kim N, Silverstein RA, da Silva JF, de la Cruz D, Richa R, Malhotra R, Chung DY, Chao LH, Tsai SQ, Maguire CA, Lindsay ME, Kleinstiver BP, Musolino PL. Alves CRR, et al. bioRxiv [Preprint]. 2024 Nov 11:2024.11.11.621817. doi: 10.1101/2024.11.11.621817. bioRxiv. 2024. PMID: 39605323 Free PMC article. Preprint.
Base editing as a genetic treatment for spinal muscular atrophy.
Alves CRR, Ha LL, Yaworski R, Lazzarotto CR, Christie KA, Reilly A, Beauvais A, Doll RM, de la Cruz D, Maguire CA, Swoboda KJ, Tsai SQ, Kothary R, Kleinstiver BP. Alves CRR, et al. bioRxiv [Preprint]. 2023 Jan 21:2023.01.20.524978. doi: 10.1101/2023.01.20.524978. bioRxiv. 2023. PMID: 36711797 Free PMC article. Preprint.
17 results