首页> 外国专利> Compositions for transfection of oligonucleotides active for gene silencing and their biological and therapeutical applications

Compositions for transfection of oligonucleotides active for gene silencing and their biological and therapeutical applications

机译:用于基因沉默的活性寡核苷酸的转染组合物及其生物学和治疗应用

摘要

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.
机译:本发明涉及转染组合物,其包含对RNA干扰具有活性的寡核苷酸和式(I)的两亲阳离子分子 <图像文件=“ IMGA0001.GIF” he =“ 45” imgContent =“ chem” imgFormat =“ GIF” wi =“ 59” /> 其中-X为N-R 1 ,S或0,R 1 为C1-C4烷基或羟基C3-C6烷基,-相同或不同的R 2 和R 3 代表H或C1-C4烷基,或R 2 和R 3 连接在一起形成一个具有6个元素的循环或杂环,-E为C1-C5烷基间隔基,-相同或不同的R 4 和R 5 表示饱和或不饱和,直链或支链的C10-C36烃或碳氟链,任选地包含C3-C6环烷基,-A -是一种生物相容性阴离子;所述组合物作为药物的用途。

著录项

相似文献

  • 专利
  • 外文文献
  • 中文文献
获取专利

客服邮箱:kefu@zhangqiaokeyan.com

京公网安备:11010802029741号 ICP备案号:京ICP备15016152号-6 六维联合信息科技 (北京) 有限公司©版权所有
  • 客服微信

  • 服务号