Restoring Protein Expression in Neuromuscular Conditions: A Review Assessing the Current State of Exon Skipping/Inclusion and Gene Therapies for Duchenne Muscular Dystrophy and Spinal Muscular Atrophy
Omar Sheikh, Toshifumi Yokota
Topics & Concepts
Spinal muscular atrophyDuchenne muscular dystrophySMA*Exon skippingMedicineDystrophinNeuromuscular diseaseMuscular dystrophyAntisense therapyMotor neuronWeaknessBioinformaticsGenetic enhancementExonInternal medicineDiseaseBiologySurgeryGeneticsGenemicroRNAAlternative splicingLocked nucleic acidCombinatoricsMathematicsNeurogenetic and Muscular Disorders ResearchMuscle Physiology and DisordersRNA Interference and Gene Delivery