Engineered AAVs for efficient noninvasive gene delivery to the central nervous system

Chan et al. (2017). Nat Neurosci DOI: 10.1038/nn.4593 Citations: 1743

Parts used

AAV-PHP.eB Serotype

Key findings

  • Developed PHP.eB through directed evolution showing 40-fold enhanced CNS transduction versus AAV9. Enabled widespread brain expression after IV injection with reduced vector dose requirements.