申请人:Grinstaff Mark W.
公开号:US20090221684A1
公开(公告)日:2009-09-03
One aspect of the present invention relates to a synthetic non-viral vector composition for gene therapy. Another aspect of the invention relates to the use of the composition for in vitro, ex vivo and/or in vivo transfer of genetic material. The invention also encompasses a pharmaceutical composition (useful for delivery of nucleic acids to a cell), containing a non-cationic amphiphilic molecule or macro-molecule; or a cationic amphiphilic molecule or macromolecule that transforms from a cationic entity to an anionic, neutral, or zwitterionic entity upon a chemical, photochemical, or biological reaction. Another aspect of the invention relates to multicationic compounds that are composed of three or more amino acids. The present invention also relates to the use of the pharmaceutical composition for delivery of nucleic acids to a cell. Moreover, the invention encompasses the non-viral vector compositions tethered to a surface. The surface-tethered compositions are useful for the delivery of nucleic acids to cells in contact with the surface. An additional embodiment of the invention relates to a hydrogel comprising a composition of the invention, and methods of using same for the delivery of genetic material to a cell.
本发明的一个方面涉及用于基因治疗的合成非病毒载体组合物。另一个方面涉及使用该组合物在体外、体内和/或体外转移遗传物质。该发明还包括一种制药组合物(用于向细胞传递核酸),其中包含非阳离子性的两性分子或大分子;或者是一个阳离子性的两性分子或大分子,在化学、光化学或生物反应中从阳离子实体转变为阴离子、中性或带电离子实体。该发明的另一个方面涉及由三个或更多氨基酸组成的多阳离子化合物。本发明还涉及使用该制药组合物向细胞传递核酸的方法。此外,该发明还涉及附着在表面上的非病毒载体组合物。附着在表面上的组合物可用于将核酸传递到与表面接触的细胞中。该发明的另一个实施例涉及包含本发明组合物的水凝胶,并使用该水凝胶将遗传物质传递给细胞的方法。