Therapeutic approaches for Duchenne muscular dystrophy
Thomas C. Roberts, Matthew J. A. Wood, Kay E. Davies
Topics & Concepts
Exon skippingDuchenne muscular dystrophyDystrophinGenome editingMedicineMuscular dystrophyBioinformaticsGenetic enhancementDrug developmentMyopathyDrug discoveryDiseaseExonComputational biologyGeneGeneticsDrugBiologyPharmacologyCRISPRAlternative splicingInternal medicineCRISPR and Genetic EngineeringAdvanced biosensing and bioanalysis techniquesRNA Interference and Gene Delivery