Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy
Adrian Pickar‐Oliver, Veronica Gough, Joel D. Bohning, Siyan Liu, Jacqueline N. Robinson-Hamm, Heather Daniels, William H. Majoros, Garth Devlin, Aravind Asokan, Charles A. Gersbach
Topics & Concepts
CRISPRGenome editingDuchenne muscular dystrophyDystrophinBiologyExonGeneticsMuscular dystrophyExon skippingGeneInsert (composites)Computational biologyAlternative splicingMechanical engineeringEngineeringCRISPR and Genetic EngineeringVirus-based gene therapy researchRNA and protein synthesis mechanisms