Assessment of rAAVrh.74.MHCK7.micro-dystrophin Gene Therapy Using Magnetic Resonance Imaging in Children With Duchenne Muscular Dystrophy
Rebecca J. Willcocks, Sean C. Forbes, Glenn A. Walter, Lee Sweeney, Louise R. Rodino‐Klapac, Jerry R. Mendell, Krista Vandenborne
Abstract
This case-control study uses magnetic resonance imaging and spectroscopy to evaluate the association between treatment with recombinant adeno-associated virus serotype rh74 (rAAVrh74) and muscle quality in children with Duchenne muscular dystrophy.
Topics & Concepts
Duchenne muscular dystrophyMagnetic resonance imagingMuscular dystrophyGenetic enhancementRecombinant DNAMedicineDystrophinGeneInternal medicineBiologyGeneticsRadiologyMuscle Physiology and DisordersVirus-based gene therapy researchViral Infections and Immunology Research