Subretinal Injection of Voretigene Neparvovec-rzyl in a Patient With RPE65-Associated Leber's Congenital Amaurosis.
Ophthalmic Surg Lasers Imaging Retina
; 50(10): 661-663, 2019 10 01.
Article
en En
| MEDLINE
| ID: mdl-31671202
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 Administration-approved 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.
Texto completo:
1
Colección:
01-internacional
Base de datos:
MEDLINE
Asunto principal:
Terapia Genética
/
Amaurosis Congénita de Leber
Tipo de estudio:
Risk_factors_studies
Límite:
Child, preschool
/
Female
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Humans
Idioma:
En
Revista:
Ophthalmic Surg Lasers Imaging Retina
Año:
2019
Tipo del documento:
Article
Pais de publicación:
Estados Unidos