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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 work, the devise a homology-independent targeted integration (HITI) strategy, which allows for robust DNA knock-in in both dividing and non-dividing cells in vitro and, more importantly, in vivo (for example, in neurons of postnatal mammals). As a proof of concept of its therapeutic potential, they demonstrate the efficacy of HITI in improving visual function using a rat model of the retinal degeneration condition retinitis pigmentosa. The HITI method presented here establishes new avenues for basic research and targeted gene therapies.