发明名称 COMPOSITIONS FOR TRANSFECTION OF OLIGONUCLEOTIDES ACTIVE FOR GENE SILENCING AND THEIR BIOLOGICAL AND THERAPEUTICAL APPLICATIONS
摘要 The invention relates to compositions of transfection comprising an oligonucleotide and an amphiphilic cationic molecule of formula (I) wherein, —X is N—R1, S or O, R1 being a C1-C4 alkyl radical or an hydroxylated C3-C6 alkyl radical, R2 and R3, identical or different, represent H or a C1-C4 alkyl radical, or R2 and R3 are linked together to form a saturated or unsaturated cycle or a heterocycle having 5 or 6 elements, E is a C1-C5 alkyl spacer, R4 and R5, identical or different, represent saturated or unsaturated, linear or branched, C10-C36 hydrocarbon or fluorocarbon chains, optionally comprising C3-C6 cycloalkyl, A- is a biocompatible anion. The invention relates to compositions active for oligonucleotides delivery into eukaryotic cells in culture, ex vivo or in vivo. The invention relates to compositions of transfection comprising an oligonucleotide active for RNA interference. Such compositions can be used as tools for biological studies or as drugs for therapies.
申请公布号 US2010048672(A1) 申请公布日期 2010.02.25
申请号 US20070226027 申请日期 2007.04.05
申请人 POLYPLUS-TRANSFECTION 发明人 NEUBERG PATRICK;BOLCATO BELLEMIN ANNE-LAURE;BEHR JEAN-PAUL;ERBACHER PATRICK
分类号 A61K48/00;A61P31/12;A61P33/00;A61P35/00;C07D233/56;C07D235/08;C07H21/04;C12N15/00 主分类号 A61K48/00
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