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Page 1
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. Among authors: kay cn. 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.
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. Among authors: kay cn. Lancet. 2024 Sep 7;404(10456):962-970. doi: 10.1016/S0140-6736(24)01447-8. Lancet. 2024. PMID: 39244273 Clinical Trial.
Tissue-specific genotype-phenotype correlations among USH2A-related disorders in the RUSH2A study.
Hufnagel RB, Liang W, Duncan JL, Brewer CC, Audo I, Ayala AR, Branham K, Cheetham JK, Daiger SP, Durham TA, Guan B, Heon E, Hoyng CB, Iannaccone A, Kay CN, Michaelides M, Pennesi ME, Singh MS, Ullah E; Foundation Fighting Blindness Consortium Investigator Group. Hufnagel RB, et al. Among authors: kay cn. Hum Mutat. 2022 May;43(5):613-624. doi: 10.1002/humu.24365. Epub 2022 Mar 21. Hum Mutat. 2022. PMID: 35266249 Free PMC article.
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. Among authors: kay cn. 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.
Residual Foveal Cone Structure in CNGB3-Associated Achromatopsia.
Langlo CS, Patterson EJ, Higgins BP, Summerfelt P, Razeen MM, Erker LR, Parker M, Collison FT, Fishman GA, Kay CN, Zhang J, Weleber RG, Yang P, Wilson DJ, Pennesi ME, Lam BL, Chiang J, Chulay JD, Dubra A, Hauswirth WW, Carroll J; ACHM-001 Study Group. Langlo CS, et al. Among authors: kay cn. Invest Ophthalmol Vis Sci. 2016 Aug 1;57(10):3984-95. doi: 10.1167/iovs.16-19313. Invest Ophthalmol Vis Sci. 2016. PMID: 27479814 Free PMC article.
48 results