Single amino acid insertion allows functional transduction of murine hepatocytes with human liver tropic AAV capsids.

ABSTRACT: Recent successes in clinical gene therapy applications have intensified the interest in using adeno-associated viruses (AAVs) as vectors for gene delivery into human liver. An inherent intriguing characteristic of AAVs is that vector variants vary substantially in their ability to transduce hepatocytes from different species. This has historically limited the value of preclinical studies using rodent models for predicting the efficiency of AAV vectors in liver-targeted gene therapy clinical studies. In this work, we aimed to investigate the key determinants of the observed differential interspecies transduction abilities among AAV variants. We took advantage of domain swapping strategies between AAV-KP1, a newly identified variant with enhanced murine liver tropism, and AAV3b, which functi

SUBMITTER: Cabanes-Creus M 

PROVIDER: S-EPMC8142051 | biostudies-literature | 2021 Jun

REPOSITORIES: biostudies-literature