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Liver gene therapy and hepatocellular carcinoma: A complex web

20 Mar 2021

Abstract excerpt

Genetic correction of inborn errors recently became a reality with the first gene therapies being approved by regulatory authorities. Several inborn errors affecting the liver have been at the forefront of gene therapy research, which often utilizes adeno-associated viral (AAV) vectors for hepatic gene transfer. Controversial evidence has been provided in humans and animal studies that wild-type AAV or, in some...

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PMID: 33743193
DOI: 10.1016/j.ymthe.2021.03.009

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