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    • 2. 发明申请
    • NOVEL SUGAR ALCOHOL-BASED COMPOSITIONS FOR DELIVERING NUCLEIC ACID-BASED DRUGS IN VIVO AND IN VITRO
    • 用于在体内和体内递送基于核酸的药物的新型基于酒石酸的组合物
    • WO2015099839A1
    • 2015-07-02
    • PCT/US2014/050114
    • 2014-08-07
    • LIN, Shi-LungWU, David TS
    • LIN, Shi-LungLIN, Yi-Wen
    • A61K47/18A61K47/26A61K9/08C12N15/113
    • A61K9/08A61K47/183A61K47/26C12N15/111C12N2310/14C12N2310/141C12N2310/3517C12N2320/32
    • This invention relates to a composition and its use for formulating nucleic acid-based drugs/vaccines with sugar alcohol compositions into complexes for both in-vitro and in-vivo delivery. Particularly, the present invention includes the ingredients and processes necessary for formulating therapeutic and pharmaceutical nucleic acid compositions, such as miRNA, microRNA precursors, shRNAs, siRNAs, ribozymes, antisense RNAs/DNAs, RNA-DNA hybrids and DNA vectors/vaccines, with glycylated sugar alcohols/sugars into delivery complexes, which can then be absorbed by cells in vivo and in vitro via active endocytosis. Also, the present invention discloses that chemical compounds containing sugar alcohol- and/or sugar-like structures can protect nucleic acids, in particular miRNAs, shRNAs, siRNAs and ribozymes, from degradation in vivo as well as in vitro. Therefore, the present invention is also a formula and method for preserving the structural integrity and functional efficacy of these nucleic acid-based drugs and/or vaccines in vivo and in vitro.
    • 本发明涉及一种组合物及其用于将糖醇组合物配制成用于体外和体内递送的复合物的核酸基药物/疫苗的用途。 特别地,本发明包括配制具有糖基化的治疗和药物核酸组合物如miRNA,微RNA前体,shRNA,siRNA,核酶,反义RNA / DNA,RNA-DNA杂交体和DNA载体/疫苗所必需的成分和过程 糖醇/糖进入递送复合物,其然后可以通过活性内吞作用在体内和体外被细胞吸收。 此外,本发明公开了含有糖醇和/或糖类结构的化合物可以保护核酸,特别是miRNA,shRNA,siRNA和核酶免受体内和体外降解。 因此,本发明也是用于在体内和体外保存这些基于核酸的药物和/或疫苗的结构完整性和功能功效的配方和方法。