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TL;DR
It concludes that while preclinical results are promising and current clinical trials are showing positive outcomes, further development is needed before these therapies can be widely translated into clinical practice.
Biodrugs · 3 authors, 3 centres
This summary was generated by AI from a single paper. It has not been reviewed by a clinician and is not clinical advice. Verify against the source before acting on it.
It concludes that while preclinical results are promising and current clinical trials are showing positive outcomes, further development is needed before these therapies can be widely translated into clinical practice.
This is a narrative review discussing adeno-associated virus (AAV)-based gene therapy strategies for retinal and choroidal vascular diseases, focusing on age-related macular degeneration and diabetic retinopathy. The authors summarize approaches from preclinical and clinical studies, including engineered vectors, multigenic constructs, tissue-specific promoters, and inducible expression systems. The review notes that preclinical studies have shown encouraging results and current clinical trials have reported promising outcomes, positioning AAV-mediated delivery as a promising approach. However, the authors highlight a key limitation: most preclinical studies assessed protective effects in healthy or pre-disease models, not in established disease settings.