A single amino acid variant in the variable region I of AAV capsid confers liver detargeting
Ruxiao Xing, Mengyao Xu, Darcy Reil, April Destefano, Mengtian Cui, Nan Liu, Jialing Liang, Guangchao Xu, Li Luo, Meiyu Xu, Fang Zhang, Phillip W.L. Tai, Yuquan Wei, Alisha M. Gruntman, Terence R. Flotte, , Dan Wang
The paper identifies an amino acid variant in AAV capsid VR-1 that reduces liver targeting, suggesting its potential for minimizing liver toxicity in gene therapy.
PLoS Pathogens · Sep 17, 2025Peer-reviewedMolecular Biology