Treatment Strategy With Gene Editing for Late-Onset Retinal Degeneration Caused by a Founder Variant in C1QTNF5.
Li RTH, Roman AJ, Sumaroka A, Stanton CM, Swider M, Garafalo AV, Heon E, Vincent A, Wright AF, Megaw R, Aleman TS, Browning AC, Dhillon B, Cideciyan AV.
Li RTH, et al.
Invest Ophthalmol Vis Sci. 2023 Dec 1;64(15):33. doi: 10.1167/iovs.64.15.33.
Invest Ophthalmol Vis Sci. 2023.
PMID: 38133503
Free PMC article.
PURPOSE: Genome editing is an emerging group of technologies with the potential to ameliorate dominant, monogenic human diseases such as late-onset retinal degeneration (L-ORD). The goal of this study was to identify disease stages and retinal location …
PURPOSE: Genome editing is an emerging group of technologies with the potential to ameliorate dominant, monogenic human diseases such as …