Large-Scale Genome Editing Based on High-Capacity Adenovectors and CRISPR-Cas9 Nucleases Rescues Full-Length Dystrophin Synthesis in DMD Muscle Cells

Fully viral gene-deleted adenovector particles were investigated as sources of optimized high-specificity CRISPR-Cas9 nucleases and donor DNA constructs tailored for targeted insertion of full-length dystrophin expression units.