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Clinical Progress and Safety Innovations in AAV Vector-Based Gene Therapy
Adeno-associated virus (AAV) vectors have secured their position as the leading viral delivery vehicle in gene therapy due to their low pathogenicity and ability to achieve stable, long-term gene expression in targeted tissues. This technology is at the forefront of treating rare genetic diseases across ophthalmic, hematological, and neurological disorders, with multiple AAV-based therapies already receiving regulatory approval. Current research is focused on refining vector design to improve delivery efficiency and address safety challenges, particularly vector dose toxicity and potential immune responses, such as transient hepatotoxicity. Scientists are actively working on novel AAV variants and enhanced purification processes to ensure the highest quality and therapeutic integrity of these life-changing treatments for patients.