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Current Clinical Applications of In Vivo Gene Therapy with AAVs.
Mendell JR, Al-Zaidy SA, Rodino-Klapac LR, Goodspeed K, Gray SJ, Kay CN, Boye SL, Boye SE, George LA, Salabarria S, Corti M, Byrne BJ, Tremblay JP. Mendell JR, et al. Mol Ther. 2021 Feb 3;29(2):464-488. doi: 10.1016/j.ymthe.2020.12.007. Epub 2020 Dec 10. Mol Ther. 2021. PMID: 33309881 Free PMC article. Review.
Intravitreal injection of a rationally designed AAV capsid library in non-human primate identifies variants with enhanced retinal transduction and neutralizing antibody evasion.
Kellish PC, Marsic D, Crosson SM, Choudhury S, Scalabrino ML, Strang CE, Hill J, McCullough KT, Peterson JJ, Fajardo D, Gupte S, Makal V, Kondratov O, Kondratova L, Iyer S, Witherspoon CD, Gamlin PD, Zolotukhin S, Boye SL, Boye SE. Kellish PC, et al. Mol Ther. 2023 Dec 6;31(12):3441-3456. doi: 10.1016/j.ymthe.2023.10.001. Epub 2023 Oct 9. Mol Ther. 2023. PMID: 37814449 Free PMC article.
Dual-AAV vector-mediated expression of MYO7A improves vestibular function in a mouse model of Usher syndrome 1B.
Lau SC, Grati M, Isgrig K, Sinan M, Calabro KR, Zhu J, Ishibashi Y, Ozgur Z, Wafa T, Belyantseva IA, Fitzgerald T, Friedman TB, Boye SL, Boye SE, Chien WW. Lau SC, et al. Mol Ther Methods Clin Dev. 2023 Aug 21;30:534-545. doi: 10.1016/j.omtm.2023.08.012. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37693946 Free PMC article.
Safety and efficacy of ATSN-101 in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D: a phase 1/2, multicentre, open-label, unilateral dose escalation study.
Yang P, Pardon LP, Ho AC, Lauer AK, Yoon D, Boye SE, Boye SL, Roman AJ, Wu V, Garafalo AV, Sumaroka A, Swider M, Viarbitskaya I, Aleman TS, Pennesi ME, Kay CN, Fujita KP, Cideciyan AV. Yang P, et al. Lancet. 2024 Sep 7;404(10456):962-970. doi: 10.1016/S0140-6736(24)01447-8. Lancet. 2024. PMID: 39244273 Clinical Trial.
Night vision restored in days after decades of congenital blindness.
Jacobson SG, Cideciyan AV, Ho AC, Roman AJ, Wu V, Garafalo AV, Sumaroka A, Krishnan AK, Swider M, Mascio AA, Kay CN, Yoon D, Fujita KP, Boye SL, Peshenko IV, Dizhoor AM, Boye SE. Jacobson SG, et al. iScience. 2022 Oct 4;25(10):105274. doi: 10.1016/j.isci.2022.105274. eCollection 2022 Oct 21. iScience. 2022. PMID: 36274938 Free PMC article.
SubILM Injection of AAV for Gene Delivery to the Retina.
Gamlin PD, Alexander JJ, Boye SL, Witherspoon CD, Boye SE. Gamlin PD, et al. Methods Mol Biol. 2019;1950:249-262. doi: 10.1007/978-1-4939-9139-6_14. Methods Mol Biol. 2019. PMID: 30783978 Free PMC article.
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