MICELLE BASED SYSTEM NUCLEASE ENCAPSULATION FOR IN-VIVO GENE EDITING

    公开(公告)号:US20190032088A1

    公开(公告)日:2019-01-31

    申请号:US16073778

    申请日:2017-02-24

    Applicant: CELLECTIS

    Abstract: The invention pertains to therapies that require gene editing, and more specifically to non-viral methods for in vivo delivery of endonuclease reagents to specific tissues or cells. According to the invention, the endonuclease reagents are encapsulated into micelle structures of 50 to 150 nm diameter for intravenous injection. The invention thus provides therapeutic composition including such micelles structures, by which endonuclease reagents can be released into cell under RNA form for their use in the treatment of gene related diseases.

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