Engineering novel AAV capsids by global de-targeting and subsequent muscle-specific tropism in mice and NHPs
Pan Yue, Yujian Zhong, Huan Chen, Youwei Zhang, Zhiyong Dai, Junlin Chen, Keqin Tan, X B Chen, Danlan Qiu, Longxiang Sheng, Xinping Tan, Ying Fan, Ye Bu, Zexin Zhou, Zhiming Yang, , Huapeng Li
The paper develops AAV.eM, a muscle-specific rAAV capsid with reduced off-target transduction, showing efficacy in Duchenne Muscular Dystrophy models and favorable safety in mice and NHPs.
bioRxiv · May 19, 2025 · PreprintBioengineering