AAV9P31 Serotype
BBB-penetrating AAV9 variant engineered through peptide-display library screening. Uses carbonic anhydrase IV receptor but shows species-limited efficacy.
Characteristics
Contains 7-amino acid insertion (WPTSYDA) in hypervariable region VIII. Uses carbonic anhydrase IV (CA-IV) receptor for BBB transcytosis. Enhanced BBB penetration in mice versus AAV9. CA-IV conservation pattern explains species-specific efficacy. Good mouse CNS transduction but limited NHP/human translation. Alternative BBB penetration mechanism to PHP series.
Applications
Mouse CNS gene therapy research. BBB transcytosis mechanism studies. Alternative to PHP variants for mouse CNS work. Demonstrates CA-IV receptor pathway for BBB crossing.
Literature References
- Deverman et al. (2018). Gene therapy for neurological disorders: progress and prospects. Nat Protoc - Deverman 2018 AAV9P31
- Hordeaux et al. (2019). The GPI-linked protein LY6A drives AAV-PHP.B transport across the blood-brain barrier. Sci Transl Med - Hordeaux 2019 CA4
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