Optimization of antisense-mediated exon skipping for Duchenne muscular dystrophy
Kasia Dzierlega, Toshifumi Yokota
Topics & Concepts
Exon skippingDuchenne muscular dystrophyExonDystrophinMuscular dystrophyMedicineBioinformaticsBiologyGeneticsGeneAlternative splicingMuscle Physiology and DisordersRNA Interference and Gene DeliveryCRISPR and Genetic Engineering