… in nonessential regions of the gene and partially restore dystrophin expression (13). This …
Similarly, adenovirus-mediated gene editing was shown to restore dystrophin expression in …

… We concluded that single-cut genomic editing using AAV9-Cas9 and AAV9-sgRNA-51
was highly efficient at restoring dystrophin expression, dystrophin-associated glycoprotein …

… Furthermore, to test if CRISPR-mediated genome editing could restore dystrophin expression
in the adult heart of dystrophic mice, we administered AAV-SaCas9/gRNAs (1×10 12 vg …

… Taken together, we show for the first time that CRISPR-mediated gene editing functionally
restores dystrophin expression in live mdx mice. This approach holds promises not only for …

… genome editing … expression of a functional dystrophin protein that is mutated in DMD.
TALENs were engineered to mediate highly efficient gene editing at exon 51 of the dystrophin gene…

… Genome editing reduced fibrosis in the cardiac muscle while immunofluorescence revealed
that dystrophin expression was restored in 11.1% ± 0.7% of cardiomyocytes. Both studies …

… Our results demonstrate the efficacy of single-cut genome editing for restoration of dystrophin
expression in a large-animal model of DMD, reaching up to ~80% of WT levels in some …

… Unfortunately, animal models recapitulating such mutations are scarce, limiting our ability
to study them and develop genome editing therapies. Here, we describe the generation of a …

… Genome editing with the CRISPR/Cas9 system has recently been used to … in gene-based
methods that restore dystrophin expression, including gene and cell therapy, genome editing, …

… In summary, this study provides evidence for the efficacy of in vivo genome editing to
correct disruptive mutations and restore dystrophin expression and function in DMD patient-derived …