Retinal transduction profiling of diverse AAV serotypes via intravitreal injection.

ABSTRACT: Optimizing adeno-associated virus (AAV) capsid and dosing selection is critical for the clinical translation of retinal gene therapy. This study aims to provide a comprehensive reference by comparing the transduction efficiency, cellular tropisms, and temporal retinal expression patterns of various AAV serotypes for intravitreal retinal gene therapy. A series of AAV vectors were intravitreally injected into C57BL/6J mice. Retinal tissues were harvested 4 weeks post-injection to evaluate the transgene expression and cellular tropisms by immunostaining. Both ssAAV2.NN and scAAV2.NN vectors at a dose escalation were administered, with similar assessments conducted at 2 and 4 weeks post-injection. Additionally, the early-phase retinal transduction profiles of AAV vectors were detected at multi

SUBMITTER: Zhang T 

PROVIDER: S-EPMC12455963 | biostudies-literature | 2025 Sep

REPOSITORIES: biostudies-literature