The GPI-linked protein LY6A drives AAV-PHP.B transport across the blood-brain barrier

Hordeaux et al. (2018). Mol Ther DOI: 10.1016/j.ymthe.2019.02.013 Citations: 258

Key findings

Identified LY6A (Ly6a in mice, SCA-1) as the specific receptor for PHP.B CNS penetration. Explained mouse strain-specificity of PHP variants. Critical for understanding PHP series limitations in non-mouse species.

Parts used

AAV-PHP.B3 Serotype AAV-PHP.B2 Serotype AAV-PHP.B1 Serotype