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Gene Therapy-Associated Uveitis (GTAU): Understanding and mitigating the adverse immune response in retinal gene therapy

14 Mar 2025

Abstract excerpt

Retinal gene therapy using adeno-associated viral (AAV) vectors has been a groundbreaking step-change in the treatment of inherited retinal diseases (IRDs) and could also be used to treat more common retinal diseases such as age-related macular degeneration and diabetic retinopathy. The delivery and expression of therapeutic transgenes in the eye is limited by innate and adaptive immune responses against...

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Share to Ocular Diseases and Behçet’s Syndrome
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PMID: 40090458
DOI: 10.1016/j.preteyeres.2025.101354

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