In Vivo Gene Editing of Muscle Stem Cells with Adeno-Associated Viral Vectors in a Mouse Model of Duchenne Muscular Dystrophy
Jennifer B. Kwon, Adarsh R. Ettyreddy, Ashish Vankara, Joel D. Bohning, Garth Devlin, Stephen D. Hauschka, Aravind Asokan, Charles A. Gersbach
Abstract
mouse.
Topics & Concepts
Duchenne muscular dystrophyIn vivoAdeno-associated virusBiologyGeneStem cellMuscular dystrophyGenome editingGenetic enhancementVirologyVector (molecular biology)Cell biologyGeneticsGenomeRecombinant DNAVirus-based gene therapy researchCRISPR and Genetic EngineeringViral Infectious Diseases and Gene Expression in Insects