Safety and Long-Term Efficacy of AAV4 Gene Therapy in Patients with RPE65 Leber Congenital Amaurosis.

ABSTRACT: The aim of this study was the evaluation of the safety and efficacy of unilateral subretinal injection of the adeno-associated vector (AAV) serotypes 2 and 4 (AAV2/4) RPE65-RPE65 vector in patients with Leber congenital amaurosis (LCA) associated with RPE65 gene deficiency. We evaluated ocular and general tolerance and visual function up to 1 year after vector administration in the most severely affected eye in nine patients with retinal degeneration associated with mutations in the RPE65 gene. Patients received either low (1.22 × 10¹⁰ to 2 × 10¹⁰ vector genomes [vg]) or high (between 3.27 × 10¹⁰ and 4.8 × 10¹⁰ vg) vector doses. An ancillary study, in which six of the original nine patients participated, extended the follow-up period to 2-3.5 yea

SUBMITTER: Le Meur G 

PROVIDER: S-EPMC5763029 | biostudies-literature | 2018 Jan

REPOSITORIES: biostudies-literature