发明名称 SYSTEMS AND METHODS FOR NUCLEIC ACID EXPRESSION IN VIVO
摘要 The present invention provides compositions, systems, kits, and methods for generating expression of one or more proteins and/or biologically active nucleic acid molecules in a subject (e.g., at therapeutic levels for extended periods required to produce therapeutic effects). In certain embodiments, systems and kits are provided that comprise a first composition comprising a first amount of polycationic structures, and a second composition comprising a therapeutically effective amount of expression vectors (e.g., non-viral expression vectors not associated with liposomes) that are CpG-free or CpG-reduced, where the expression vectors comprise a first nucleic acid sequence encoding: i) a first therapeutic protein or proteins, and/or ii) a first biologically active nucleic acid molecule or molecules.
申请公布号 WO2017049132(A1) 申请公布日期 2017.03.23
申请号 WO2016US52205 申请日期 2016.09.16
申请人 DNARX 发明人 DEBS, Robert James;HEATH, Timothy D.;HANDUMRONGKUL, Chakkrapong
分类号 A61K9/127;A61K31/573;A61K48/00;C12N15/63;C12N15/64 主分类号 A61K9/127
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