Decoupling Efficacy from Toxicity: Engineering Spatial Control in AAV-Mediated Gene Therapy
Ying Fan, Keqin Tan, Huan Chen, Xiaoqu Chen, Yue Pan, Yajing Chen, Yangsiqi Ao, Ye Bu, Huapeng Li
This study shows that using tissue-specific AAV variants and local delivery reduces hepatic toxicity while maintaining therapeutic efficacy in gene therapy.
bioRxiv · Dec 26, 2025 · PreprintCancer Biology