A comprehensive study of AAV tropism across C57BL/6 mice, BALB/c mice, and crab-eating macaques.

ABSTRACT: Recombinant adeno-associated viruses (AAVs) have been widely used for gene delivery and gene therapy. However, certain AAV serotypes exhibited distinct transduction patterns among different mouse strains or between mice and non-human primates (NHPs). These variations prompted us to investigate the AAV tropism of 21 capsid variants using barcoded AAV libraries among different tissues in C57BL/6 and BALB/c mice, as well as in crab-eating macaques. Our study unveiled that AAV tropisms varied significantly among different mouse strains and species, particularly in capsid variants such as AAV4, AAV9, PHP.B, and CAP-B10. Notably, AAV4 exhibited liver-detargeting properties in both mice and NHPs, and was remarkably efficient in transducing the lung, glomerulus, and pancreatic islet. These finding

SUBMITTER: Fang K 

PROVIDER: S-EPMC11919325 | biostudies-literature | 2025 Mar

REPOSITORIES: biostudies-literature