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Article

Liver Gene Therapy: Reliable and Durable?

17 Oct 2019

Abstract excerpt

The liver is an important target for gene therapy for metabolic diseases and systemic delivery of therapeutic proteins. The vast majority of our knowledge on clinical gene therapy directed to the liver comes from treatment of hemophilia A and B with adeno-associated viral (AAV) vectors. This approach relies on hepatic tropism of the viral capsid following intravenous infusion of the vector. Interestingly, early...

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Identifiers

PMID: 31630963
DOI: 10.1016/j.ymthe.2019.10.005

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