High-efficiency transduction and correction of murine hemophilia B using AAV2 vectors devoid of multiple surface-exposed tyrosines

Markusic et al. (2010). Mol Ther DOI: 10.1038/mt.2010.172 Citations: 175

Key findings

AAV8BP2 is a bioprocessing-optimized AAV8 variant with enhanced manufacturability. Improved thermal stability and higher production yields. Maintained wild-type AAV8 tropism profile while enabling scalable clinical manufacturing.