AAV-PHP.B1 Serotype

First-generation blood-brain barrier penetrating AAV variant evolved from AAV9 using CREATE methodology. Mouse strain-specific due to LY6A receptor dependence.

Length: 1 bp

Tropism: CNS (widespread), peripheral nervous system (mouse-specific)

Characteristics

Seven-amino acid insertion (TLAVPFK) in capsid position 588. 40-fold enhanced BBB penetration versus AAV9 in C57BL/6 mice. LY6A receptor-dependent CNS entry. Mouse strain-specific efficacy. Limited translation to primates.

Applications

CNS gene therapy in mouse models. Neurological disease research. Circuit mapping tools. Proof-of-concept for BBB-penetrating AAV engineering.

Literature References

  1. Deverman et al. (2016). Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain. Nat Biotechnol - Deverman 2016 PHP.B
  2. Hordeaux et al. (2018). The GPI-linked protein LY6A drives AAV-PHP.B transport across the blood-brain barrier. Mol Ther - Hordeaux 2018 LY6A

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