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    Home > Wang Ming research group of Institute of chemistry, Chinese Academy of Sciences develops CRISPR / cas9 delivery and new strategy of in vivo gene editing

    Wang Ming research group of Institute of chemistry, Chinese Academy of Sciences develops CRISPR / cas9 delivery and new strategy of in vivo gene editing

    • Last Update: 2020-01-30
    • Source: Internet
    • Author: User
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    CRISPR / cas9 is a gene editing technology derived from the acquired immune system of bacteria, which has a wide application prospect in the basic research of chemical biology and the development of new gene therapy technology However, one of the key problems in biomedical applications is to deliver cas9 nuclease with gene editing function into cells and realize gene editing in vivo Recently, with the support of NSFC, the Ministry of science and technology and the Chinese Academy of Sciences, Wang Ming's research group has designed the molecular self-assembly between cationic liposomes and cas9 messenger RNA (cas9 mRNA) to prepare cas9 mRNA nanoparticles, which are further applied to CRISPR / cas9 delivery and in vivo gene editing The researchers first designed the cationic liposome carrier modified by phenylboric acid, and realized the cell line selective cas9 mRNA delivery and targeted gene editing (ACS Applied Materials & interfaces, 2019, 11, 46585-46590) 。 On the basis of the above, the author further designed a cationic liposome carrier which can be degraded in cell microenvironment, and realized efficient cas9 mRNA delivery and in vivo gene editing The results show that the above-mentioned mRNA nanoparticles can degrade under the action of reduced glutathione, release cas9 mRNA and translate cas9 nuclease for gene editing Using liposome bamea-o16b to deliver cas9 mRNA can effectively edit the expression of report signal protein (the editing efficiency of GFP gene is as high as 90%), while bamea-o16b / cas9 mRNA nanoparticles injected through tail vein can be enriched in mouse liver parenchyma cells and regulate the pre protein converting enzyme kexin9 (PCSK9 , a gene related to LDL and cholesterol metabolism in vivo Single injection of bamea-o16b / cas9 mRNA nanoparticles reduced PCSK9 protein level in mice serum by more than 80% Relevant research results were recently published in adv.mater (adv.mater 2019, 31, 1902575) Ph.D student Liu Ji as the first author After the publication of the paper, it was also reported by xinhuanet.com as a research highlight.
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