Retinal gene therapy using epiretinal AAV-containing fibrin hydrogel implants.

ABSTRACT: Subretinal injection of adeno-associated virus (AAV) is generally more efficacious and less inflammatory than intravitreal injection for retinal gene therapy. However, adverse events (e.g., chorioretinal atrophy) have been reported in many patients receiving subretinal injection of Luxturna (voretigene neparvovec-rzyl) and experimental gene therapies. Subretinal injection confines transduction to the treated area. To address this, we manufactured high-concentration fibrin hydrogels encapsulating AAV2-green fluorescent protein (AAV2-GFP). Gels had homogeneous AAV distribution, desired mechanical properties, and retained infectivity. Epiretinal placement of fibrin-AAV2-GFP (n = 11) was compared to subretinal (n = 5) and intravitreal AAV2-GFP injection (n

SUBMITTER: Scruggs BA 

PROVIDER: S-EPMC12412667 | biostudies-literature | 2025 Sep

REPOSITORIES: biostudies-literature