Genetic Engineering Publications - GEG Tech top picks

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.