Potential limitations of micro-dystrophin gene therapy for Duchenne muscular dystrophy
C. C. Hart, Y. i. Lee, J. Xie, G. Gao, D. W. Hammers, L. Sweeney
This study evaluates micro-dystrophin gene therapy in a DMD mouse model, finding cardiac disease acceleration with one micro-dystrophin and potential benefits with another, highlighting the need for careful dosing.
bioRxiv · Oct 2, 2022Pharmacology & Toxicology