Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain

Deverman et al. (2016). Nat Biotechnol DOI: 10.1038/nbt.3440 Citations: 1277

Key findings

Engineered PHP.B through CREATE (Cre recombination-based AAV targeted evolution) from AAV9. Seven-amino acid insertion (TLAVPFK) in capsid. Achieved >40-fold enhanced BBB penetration versus AAV9 in mice. First demonstration of rational CNS-directed AAV evolution.

Parts used

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