首页> 外国专利> IMPROVEMENTS IN ANTISENSE OLIGOMERS, DELIVERY OF ANTISENSE OLIGOMERS, AND IDENTIFICATION OF ANTISENSE OLIGOMER TARGETS

IMPROVEMENTS IN ANTISENSE OLIGOMERS, DELIVERY OF ANTISENSE OLIGOMERS, AND IDENTIFICATION OF ANTISENSE OLIGOMER TARGETS

机译:反义寡聚物的改进,反义寡聚物的交付以及反义寡聚物目标的确定

摘要

Antisense oligomers which possess improved properties over those taught in the prior art are disclosed. Preferably, oligomers for delivery to a cell comprising at least one nucleomonomer having a silicon-based 2'OH protecting group, e.g., a tertbutyl dimethyl-silyl group. Oligomers of between about 7 and about 12 nucleomonomers in length which are complementary to a site on an RNA target molecule selected from the group consisting of: the extreme 5' terminus of the RNA molecule, a 5' splice junction of the RNA molecule, and a 3' splice junction of the RNA molecule are provided. Oligomers comprising a neutral backbone which have been modified to comprise at least one charged group are also disclosed. In addition, methods of identifying an oligomer complementary to a cellular gene involved in a physiological process are provided.
机译:公开了反义低聚物,其具有比现有技术教导的那些更高的性能。优选地,用于递送至细胞的低聚物包括至少一种具有基于硅的2'OH保护基,例如叔丁基二甲基-甲硅烷基的核单体。长约7至约12个核单体的寡聚体,其与RNA靶分子上选自以下的组的位点互补:RNA分子的极端5'末端,RNA分子的5'剪接点和提供了RNA分子的3'剪接连接。还公开了包含中性主链的低聚物,其已被修饰以包含至少一个带电基团。另外,提供了鉴定与参与生理过程的细胞基因互补的寡聚物的方法。

著录项

  • 公开/公告号EP1206532A2

    专利类型

  • 公开/公告日2002-05-22

    原文格式PDF

  • 申请/专利权人 SEQUITUR INC.;TRILINK BIOTECHNOLOGIES INC.;

    申请/专利号EP20000950992

  • 发明设计人 WOOLF TOD M.;BECK TERRY B.;

    申请日2000-08-03

  • 分类号C12N15/11;C07H21/00;C12Q1/68;A61K31/712;C12N15/87;

  • 国家 EP

  • 入库时间 2022-08-22 00:33:21

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