Safety and Efficacy of Adeno-Associated Viral Gene Therapy in Patients With Retinal Degeneration: A Systematic Review and Meta-Analysis.

ABSTRACT:

Purpose

This systematic review evaluates the safety and efficacy of ocular gene therapy using adeno-associated virus (AAV).

Methods

MEDLINE, Embase, Cochrane Central Register of Controlled Trials, and ClinicalTrials.gov were searched systematically for controlled or non-controlled interventional gene therapy studies using key words related to retinal diseases, gene therapy, and AAV vectors. The primary outcome measure was safety, based on ocular severe adverse events (SAEs). Secondary outcome measures evaluated efficacy of the therapy based on best corrected visual acuity (BCVA) and improvements in visual sensitivity and systemic involvement following ocular delivery. Pooling was done using a DerSimonian Laird random effects model. Risk of bias was assessed using the Cochran

SUBMITTER: Sobh M 

PROVIDER: S-EPMC10668613 | biostudies-literature | 2023 Nov

REPOSITORIES: biostudies-literature