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.