Development of In Vitro Potency Assay for AAV-mediated Gene Therapy for Muscle Disorders

This novel vitro, cell‑based potency assay for evaluating adeno‑associated virus (AAV)–mediated gene therapies targeting muscle disorders, particularly those utilizing AAV9 vectors. This represents a significant improvement in potency determination workflows, providing a reproducible, scalable, and regulatory‑relevant alternative to animal‑based assays for muscle‑directed gene therapies.

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