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Novel vectors and approaches for gene therapy in liver diseases

30 Apr 2021

Abstract excerpt

Gene therapy is becoming an increasingly valuable tool to treat many genetic diseases with no or limited treatment options. This is the case for hundreds of monogenic metabolic disorders of hepatic origin, for which liver transplantation remains the only cure. Furthermore, the liver contains 10-15% of the body's total blood volume, making it ideal for use as a factory to secrete proteins into the circulation. In...

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PMID: 34159305
DOI: 10.1016/j.jhepr.2021.100300

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