PhysiologybioRxiv
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…·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.
DOI 10.1126/sciadv.abn0379