AAV-Mediated Dual-Gene Therapy Restores Metabolic Function in Mice with Propionic Acidemi
Hui Xu, Zewen Tao, T. Zhang, Xuli Zhang, Ying Zhou, Ziyan Cen, Jie Liu, Hao Zhang, Alimujiang Maimaitijiang, Dan Chen, Dali Li, Shuming Yin, Lei An, Xinwen Huang, Yue Zhang
This study demonstrates that dual-gene AAV therapy restores metabolic function and shows long-term safety in a mouse model of propionic acidemia.
bioRxiv · Mar 7, 2026 · PreprintGenetics