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Current Clinical Applications of In Vivo Gene Therapy with AAVs

10 Dec 2020

Abstract excerpt

Hereditary diseases are caused by mutations in genes, and more than 7,000 rare diseases affect over 30 million Americans. For more than 30 years, hundreds of researchers have maintained that genetic modifications would provide effective treatments for many inherited human diseases, offering durable and possibly curative clinical benefit with a single treatment. This review is limited to gene therapy using...

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PMID: 33309881
DOI: 10.1016/j.ymthe.2020.12.007

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