Evolving Therapeutic Options for the Treatment of Duchenne Muscular Dystrophy
Eleonora S. D’Ambrosio, Jerry R. Mendell
Topics & Concepts
Duchenne muscular dystrophyExon skippingDystrophinMedicineCRISPRGenetic enhancementMuscular dystrophyBioinformaticsDiseaseGenome editingExonGeneGeneticsBiologyPathologyInternal medicineAlternative splicingMuscle Physiology and DisordersNeurogenetic and Muscular Disorders ResearchVirus-based gene therapy research