Correction of Three Prominent Mutations in Mouse and Human Models of Duchenne Muscular Dystrophy by Single-Cut Genome Editing
Yi-Li Min, Francesco Chemello, Hui Li, Cristina Rodríguez-Caycedo, Efrain Sanchez‐Ortiz, Alex A. Mireault, John McAnally, John M. Shelton, Yu Zhang, Rhonda Bassel‐Duby, Eric N. Olson
Topics & Concepts
DystrophinDuchenne muscular dystrophyGenome editingBiologyExonExon skippingMuscular dystrophyCRISPRGeneticsmdx mouseCas9GeneComputational biologyBioinformaticsAlternative splicingCRISPR and Genetic EngineeringRNA Interference and Gene DeliveryRNA and protein synthesis mechanisms