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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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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