The DMD gene and therapeutic approaches to restore dystrophin
F. Fortunato, Marianna Farnè, Alessandra Ferlini
Topics & Concepts
DystrophinDuchenne muscular dystrophyExon skippingMedicineMuscular dystrophyBioinformaticsDiseaseMyopathyPhenotypeWeaknessGeneticsExonGeneBiologyPathologyInternal medicineAlternative splicingAnatomyMuscle Physiology and DisordersCRISPR and Genetic EngineeringRNA Interference and Gene Delivery