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Liver directed adeno‐associated viral vectors to treat metabolic disease

31 May 2023

Abstract excerpt

The liver is the metabolic center of the body and an ideal target for gene therapy of inherited metabolic disorders (IMDs). Adeno-associated viral (AAV) vectors can deliver transgenes to the liver with high efficiency and specificity and a favorable safety profile. Recombinant AAV vectors contain only the transgene cassette, and their payload is converted to non-integrating circular double-stranded DNA episomes,...

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PMID: 37254440
DOI: 10.1002/jimd.12637

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