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Current Clinical Applications of in vivo Gene Therapy with AAVs

Mendell JR, Al-Zaidy SA, Rodino-Klapac LR, et al. Current Clinical Applications of in vivo Gene Therapy with AAVs. Molecular therapy : the journal of the American Society of Gene Therapy 2020   Abstract Hereditary diseases are caused by mutations in genes, and more than 7,000 rare diseases affect over 30 million Americans. Since more than […]