Leber Hereditary Optic Neuropathy Gene Therapy: Adverse Events and Visual Acuity Results of All Patient Groups.

ABSTRACT:

Purpose

To assess safety of gene therapy in G11778A Leber hereditary optic neuropathy (LHON).

Design

Phase 1 clinical trial.

Methods

Setting: single institution.

Participants

Patients with G11778A LHON and chronic bilateral visual loss >12 months (group 1, n = 11), acute bilateral visual loss <12 months (group 2, n = 9), or unilateral visual loss (group 3, n = 8).

Intervention

unilateral intravitreal AAV2(Y444,500,730F)-P1ND4v2 injection with low, medium, high, and higher doses to worse eye for groups 1 and 2 and better eye for group 3.

Outcome measures

Best-corrected visual acuity (BCVA), adverse events, and vector antibody responses. Mean follow-up was 24 months (range, 12-36 months); BCVAs were compared with a published prospective natural hist

SUBMITTER: Lam BL 

PROVIDER: S-EPMC9444871 | biostudies-literature | 2022 Sep

REPOSITORIES: biostudies-literature