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Article

Immune Responses to Muscle-Directed Adeno-Associated Viral Gene Transfer in Clinical Studies

1 May 2023

Abstract excerpt

Muscle-directed gene therapy with adeno-associated viral (AAV) vectors is undergoing clinical development for treating neuromuscular disorders and for systemic delivery of therapeutic proteins. Although these approaches show considerable therapeutic benefits, they are also prone to induce potent immune responses against vector or transgene products owing to the immunogenic nature of the intramuscular delivery...

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PMID: 37154743
DOI: 10.1089/hum.2023.056

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