Pharmacology and toxicology of eteplirsen and SRP-5051 for DMD exon 51 skipping: an update
Omar Sheikh, Toshifumi Yokota
Topics & Concepts
Exon skippingMedicineDuchenne muscular dystrophyMorpholinoPharmacologyInternal medicineBiologyExonGeneticsGeneZebrafishAlternative splicingMuscle Physiology and DisordersRNA Interference and Gene DeliveryTelomeres, Telomerase, and Senescence