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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: massengill mt. 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.
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