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Genetic Engineering Publications - GEG Tech top picks
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In this study, the authors present here the CRISPR-induced deletion (CinDel), a new promising genome-editing technology to correct the DMDgene. Using an adequate pair of gRNAs, the deletion of parts of these exons and the intron separating them restored the DMDreading frame in 62% of the hybrid exons in vitro in DMD myoblasts and in vivo in electroporated hDMD/mdx mice. Given that CinDel induces permanent reparation of the DMDgene, this treatment would not have to be repeated as it is the case for exon skipping induced by oligonucleotides. 
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In this study, the scientists deployed Cpf1 to correct DMD mutations in patient-derived induced pluripotent stem cells (iPSCs) and mdx mice, an animal model of DMD. Cpf1-mediated genomic editing of human iPSCs, either by skipping of an out-of-frame DMD exon or by correcting a nonsense mutation, restored dystrophin expression after differentiation to cardiomyocytes and enhanced contractile function. Similarly, pathophysiological hallmarks of muscular dystrophy were corrected in mdx mice following Cpf1-mediated germline editing. These findings are the first to show the efficiency of Cpf1-mediated correction of genetic mutations in human cells and an animal disease model and represent a significant step toward therapeutic translation of gene editing for correction of DMD.