首页> 外国专利> TRANSFECTION PARTICLES FOR DELIVERY OF NUCLEIC ACID INTO HIGHER EUCARYOTIC CELLS I(IN VITRO) AND I(IN VIVO) COMPRISES ONE OR MORE NUCLEIC ACID, PHARMACEUTICAL COMPOSITION COMPRISING THEM AND A KIT

TRANSFECTION PARTICLES FOR DELIVERY OF NUCLEIC ACID INTO HIGHER EUCARYOTIC CELLS I(IN VITRO) AND I(IN VIVO) COMPRISES ONE OR MORE NUCLEIC ACID, PHARMACEUTICAL COMPOSITION COMPRISING THEM AND A KIT

机译:用于将核酸转运到高级(体外)和体内(体内)的核酸的翻译颗粒,包含一种或多种核酸,包含它们的药物组合物和一个试剂盒

摘要

Transfection particles for delivery of nucleic acid into higher eucaryotic cells in vitro and in vivo comprises one or more nucleic acid molecules condensed by organic cationic molecules. The discrete and stable particles are obtained by complexing the nucleic acid molecules with identical or different organic cationic precursor molecules without crosslinking nucleic acid molecules, and covalently linking the precursor molecules to each other on the nucleic acid template. For specific cellular targeting, the particles may carry targeting molecules, e.g. sugars. Preferred cationic precursor molecules are lipophilic detergents that are linked to form lipids. The particles contain preferably only one nucleic acid molecule which makes them useful for gene therapy and for delivery of large DNA molecules. IMAGE
机译:用于在体外和体内将核酸递送到高等真核细胞中的转染颗粒包含被有机阳离子分子缩合的一种或多种核酸分子。通过使核酸分子与相同或不同的有机阳离子前体分子络合而不交联核酸分子,并在核酸模板上将前体分子彼此共价连接,从而获得离散且稳定的颗粒。对于特定的细胞靶向,所述颗粒可以携带靶向分子,例如分子。糖。优选的阳离子前体分子是连接形成脂质的亲脂性去污剂。颗粒优选仅包含一个核酸分子,这使其可用于基因治疗和大DNA分子的递送。 <图像>

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