Results at 5 Years After Gene Therapy for RPE65-Deficient Retinal Dystrophy.

ABSTRACT: Previously, results at 2 years after subretinal injection of a recombinant adeno-associated virus vector expressing RPE65 (rAAV2-CB-hRPE65) in eight adults and four children with retinal degeneration caused by RPE65 mutations were reported. Now, results at 5 years after treatment in 11 of these subjects are reported. Subjects received a subretinal injection of rAAV2-CB-hRPE65 in the poorer-seeing eye, at either of two dose levels, and were followed for 5 years after treatment. The primary safety outcomes were ocular and non-ocular adverse events. Efficacy outcomes included changes in best corrected visual acuity, static perimetry hill of vision measurements for the central 30° (V₃₀), and total (V_TOT) visual field and kinetic perimetry visual field

SUBMITTER: Pennesi ME 

PROVIDER: S-EPMC12199623 | biostudies-literature | 2018 Dec

REPOSITORIES: biostudies-literature