Large1 Gene Transfer in Older myd Mice with Severe Muscular Dystrophy Restores Muscle Function and Greatly Improves Survival
T. Yonekawa, A. J. Rauckhorst, S. Y. El-Hattab, M. A. Cuellar, D. Venzke, M. E. Anderson, H. Okuma, A. D. Pewa, E. B. Taylor, K. P. Campbell
This study shows that gene transfer using AAV2/9 CMV Large1 in older myd mice with severe muscular dystrophy restores muscle function and significantly improves survival.
bioRxiv · Oct 28, 2021Physiology