T2, MTR, and diffusion tensor quantitative MRI methods reliably monitor AAV-mediated gene therapy treatment in a murine muscular dystrophy model

Herein we investigate the capability of multi-parametric MRI to reliably and decisively monitor both minute qualitative and quantitative changes in the skeletal muscle microenvironment in a Duchenne muscular dystrophy mouse model, namely mdx^4Cv. Through the use of an assortment of pulse sequences, including T2, magnetization transfer ratio, and diffusion tensor imaging, the progression of a recombinant adeno-associated viral vector treatment, by the delivery of a truncated micro-dystrophin gene, is monitored in these mice and better understood. This study fundamentally seeks to understand and confirm the practicality of MRI as a diagnostic and prognostic measure in muscular dystrophy models.