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Genetic Engineering Publications - GEG Tech top picks
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BigField GEG Tech's insight:
In this report, the authors used the CRISPR/Cas9 system to integrate a transgene in the AAVS1 locus. They observed that the knock-in of AAVS1 has significantly reduced MBS85 expression and increased contractile force. These results should be take in account in the design of gene therapy strategy which involve knock-in of AAVS1. 
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Scoop.it!
BigField GEG Tech's insight:
In this study, the scientists report the use of CRISPR/Cas9 technology to target a specific genomic region in human pluripotent stem cells. They show that CRISPR/Cas9 can be used to disrupt a gene by introducing frameshift mutations to gene coding region; to knock in specific sequences, to targeted genomic locus via homology directed repair and to induce large genomic deletion through dual-guide multiplex.
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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.