Compositions for transfection of oligonucleotides active for gene silencing and their biological and therapeutical applications
申请人:Polyplus-Transfection SA
公开号:EP1844772A1
公开(公告)日:2007-10-17
The invention relates the compositions of transfection comprising an oligonucleotide active for RNA interference and an amphiphilic cationic molecule of formula (I)
wherein
- X is N-R1, S or 0, 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 cycle or a heterocycle having 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 cycloalkyle,
- A- is a biocompatible anion.
Use of said compositions as drugs.
该发明涉及包含用于RNA干扰的寡核苷酸活性和具有以下结构的两性阳离子分子的转染组合物:其中- X为N-R1、S或O,其中R1是C1-C4烷基基团或羟基化的C3-C6烷基基团,- R2和R3,相同或不同,代表H或C1-C4烷基基团,或者R2和R3连接在一起形成具有6个元素的环或杂环,- E是C1-C5烷基间隔,- R4和R5,相同或不同,代表饱和或不饱和的线性或支链的C10-C36烃基或氟烃基链,可选地包括C3-C6环烷基,- A-是生物相容性阴离子。将该组合物用作药物。