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Mutation-independent rhodopsin gene therapy by knockdown and replacement with a single AAV vector.
Cideciyan AV, Sudharsan R, Dufour VL, Massengill MT, Iwabe S, Swider M, Lisi B, Sumaroka A, Marinho LF, Appelbaum T, Rossmiller B, Hauswirth WW, Jacobson SG, Lewin AS, Aguirre GD, Beltran WA. Cideciyan AV, et al. Among authors: sudharsan r. Proc Natl Acad Sci U S A. 2018 Sep 4;115(36):E8547-E8556. doi: 10.1073/pnas.1805055115. Epub 2018 Aug 20. Proc Natl Acad Sci U S A. 2018. PMID: 30127005 Free PMC article.
Gene therapy reforms photoreceptor structure and restores vision in NPHP5-associated Leber congenital amaurosis.
Aguirre GD, Cideciyan AV, Dufour VL, Ripolles-García A, Sudharsan R, Swider M, Nikonov R, Iwabe S, Boye SL, Hauswirth WW, Jacobson SG, Beltran WA. Aguirre GD, et al. Among authors: sudharsan r. Mol Ther. 2021 Dec 1;29(12):3528. doi: 10.1016/j.ymthe.2021.10.021. Epub 2021 Oct 29. Mol Ther. 2021. PMID: 34715016 Free PMC article. No abstract available.
18 results