Abstract
Leber’s congenital amaurosis (LCA) is a rare inherited retinal degeneration (IRD) that causes severe vision loss, nyctalopia, and nystagmus within the first few years of life. RPE65 gene mutations cause approximately 6% of LCA cases and have become the target for therapy since voretigene neparvovec-rzyl became the first U.S. Food and Drug Administrationapproved gene therapy product for IRDs in 2017. The surgery involves pars plana vitrectomy with subretinal injection of a viral vector that carries a functional copy of the RPE65 gene. Intraoperative optical coherence tomography is a useful adjunctive tool to confirm the injection has reached the subretinal space.
Original language | English (US) |
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Pages (from-to) | 661-663 |
Number of pages | 3 |
Journal | Ophthalmic Surgery Lasers and Imaging Retina |
Volume | 50 |
Issue number | 10 |
DOIs | |
State | Published - 2019 |
ASJC Scopus subject areas
- Surgery
- Ophthalmology