LIPOPOLYAMINES AS TRANSFECTION AGENTS AND PHARMACEUTICAL USES THEREOF

摘要

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.