SiRNA delivery system compound as well as preparation method and application thereof

    公开(公告)号:US12194145B2

    公开(公告)日:2025-01-14

    申请号:US17227408

    申请日:2021-04-12

    Abstract: The invention relates to the technical field of gene treatment and nano targeted delivery, in particular to a siRNA delivery system compound as well as a preparation method and application thereof. The preparation method comprises the following steps of reacting Boc-protected histidine with cholesterol to generate Boc-protected histidine cholesteryl ester, and removing BOC-released protected amino in trifluoroacetic acid to generate histidine cholesteryl ester; then, enabling amino to react with carboxyl of carboxymethyl chitosan to modify long-chain carboxymethyl chitosan to generate carboxymethyl chitosan modified by histidine cholesteryl ester, and performing targeted labeling on carboxymethyl chitosan modified by histidine cholesteryl ester to generate a carboxymethyl chitosan high-molecular compound modified by histidine cholesteryl ester and a targeted marker together, then forming a stable spherical delivery vector through self-assembly above critical micelle concentration, and mixing the stable spherical delivery vector with siRNA (small interfering ribonucleic acid) to obtain the siRNA delivery system compound.

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