Non-viral delivery of nucleic acid for treatment of rare diseases of the muscle
Divya Rao, Munia Ganguli
Topics & Concepts
Spinal muscular atrophyGenetic enhancementMedicineDuchenne muscular dystrophyMyopathyMuscle disorderMuscular dystrophyExon skippingBioinformaticsViral vectorWeaknessDiseaseBiologyGeneGeneticsPathologyExonInternal medicineAnatomyRecombinant DNAAlternative splicingMuscle Physiology and DisordersRNA Interference and Gene DeliveryCRISPR and Genetic Engineering