Yuhei Ashida
Senior Scientist JCR Pharmaceuticals Co Ltd
Seminars
Wednesday 9th December 2026
Proof-of-Concept for CNS Gene Therapy Using JUST-AAV, a Novel Tissue-Specific AAV Vector
9:00 am
- By incorporating tissue-specific binders into the AAV capsid, we have successfully engineered JUST-AAV vectors with enhanced tissue selectivity
- Introduction of targeted mutations on the capsid surface has significantly reduced off-target transduction in non-target tissues such as the liver
- JUST-AAV vectors have demonstrated high therapeutic efficacy in multiple mouse models of central nervous system diseases
