Dystrophin Gene-Editing Stability Is Dependent on Dystrophin Levels in Skeletal but Not Cardiac Muscles
Niclas E. Bengtsson, Hichem Tasfaout, Stephen D. Hauschka, Jeffrey S. Chamberlain
Topics & Concepts
DystrophinDuchenne muscular dystrophySkeletal muscleBiologyGenome editingMuscular dystrophyExonExon skippingGuide RNARegeneration (biology)mdx mouseCell biologyCRISPRGeneticsMolecular biologyGeneAlternative splicingEndocrinologyCRISPR and Genetic EngineeringVirus-based gene therapy researchMuscle Physiology and Disorders