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Sugar alcohol-based compositions for delivering nucleic acid-based drugs in vivo and in vitro

机译:基于糖醇的组合物,用于在体内和体外递送基于核酸的药物

摘要

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.
机译:本发明涉及一种组合物及其将糖醇组合物的基于核酸的药物/疫苗配制成复合物用于体外和体内递送的用途。特别地,本发明包括配制成糖基化的治疗和药物核酸组合物,例如miRNA,微小RNA前体,shRNA,siRNA,核酶,反义RNA / DNA,RNA-DNA杂交体和DNA载体/疫苗的必需成分和方法。糖醇/糖制成递送复合物,然后可通过主动内吞作用在体内和体外被细胞吸收。另外,本发明公开了含有糖醇和/或糖样结构的化合物可以保护核酸,特别是miRNA,shRNA,siRNA和核酶,在体内和体外均不降解。因此,本发明还是用于在体内和体外保持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。

著录项

  • 公开/公告号US9387251B2

    专利类型

  • 公开/公告日2016-07-12

    原文格式PDF

  • 申请/专利权人 SHI-LUNG LIN;DAVID T S WU;

    申请/专利号US201414457829

  • 发明设计人 SHI-LUNG LIN;YI-WEN LIN;

    申请日2014-08-12

  • 分类号A61K47/26;A61K31/713;A61K31/7105;C12N15/11;C12N15/113;

  • 国家 US

  • 入库时间 2022-08-21 14:33:28

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