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    • 25. 发明申请
    • LIPOPOLYAMINES AS TRANSFECTION AGENTS AND PHARMACEUTICAL USES THEREOF
    • 作为转移剂及其药物用途的脂多糖
    • WO1996017823A1
    • 1996-06-13
    • PCT/FR1995001595
    • 1995-12-04
    • RHONE-POULENC RORER S.A.BYK, GerardoDUBERTRET, CatherineSCHERMAN, Daniel
    • RHONE-POULENC RORER S.A.
    • C07C235/06
    • C12N15/88A61K47/54A61K47/543A61K48/00C07C233/05C07C235/06C07J41/0055
    • Cationic lipids of general formula (I), wherein m is an integer from 2 to 6 inclusive; n is an integer from 1 to 9 inclusive, preferably 1-5, where, when n is 2-9, a single R grouping other than hydrogen is present in the general formula, and m has variable or identical values within the groupings (a) or -(CH2)m-; R is a hydrogen atom or a radical of general formula (II), wherein X and X', which are the same or different, are an oxygen atom, a methylene grouping -(CH2)q- where q is 0, 1, 2 or 3, or an amino grouping -NH- or -NR'-, where R' is a C1-4 alkyl grouping; Y and Y', which are the same or different, are a methylene grouping, a carbonyl grouping or a C=S grouping; R3, R4 and R5, which are the same or different, are a hydrogen atom or an optionally substituted C1-4 alkyl radical, and p is 0-5; and R6 is a cholesterol derivative or an alkylamino grouping -NR1R2, where R1 and R2 are, independently of each other, a straight or branched, saturated or unsaturated C12-22 aliphatic radical. Pharmaceutical compositions containing said lipids, and their uses for transfecting nucleic acids whether in vitro or in vivo in cells, are also disclosed.
    • 通式(I)的阳离子脂质,其中m为2〜6的整数; n为1〜9的整数,优选为1〜9,其中,当n为2-9时,在通式中存在除氢以外的单个R基团,m在分组内具有可变或相同的值(a )或 - (CH 2)m - ; R是氢原子或通式(II)的基团,其中X和X'相同或不同,是氧原子,亚甲基 - (CH 2)q - ,其中q是0,1,2 或3,或氨基-NH-或-NR'-,其中R'为C 1-4烷基; Y和Y'相同或不同,是亚甲基,羰基或C = S基团; R 3,R 4和R 5相同或不同,为氢原子或任选取代的C 1-4烷基,p为0-5; R6为胆固醇衍生物或烷基氨基-NR1R2,其中R1和R2彼此独立地为直链或支链的饱和或不饱和的C 12-22脂族基。 还公开了含有所述脂质的药物组合物及其用于转染核酸的用途,无论是体外还是体内细胞。