ROZEMA, DAVID B.,罗兹马 大卫B,羅茲馬 大衛B,WAKEFIELD, DARREN H.,维克菲尔德 戴伦H,維克菲爾德 戴倫H,ALMEIDA, LAUREN J.,阿梅塔 罗伦J,阿梅塔 羅倫J
申请号：
TW105108327
公开号：
TW201639962A
申请日:
2016.03.17
申请国别(地区):
TW
年份:
2016
代理人:
摘要：
Described are RNAi interference agents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to double stranded modified oligonucleotide molecules having blunt ends and a least one ribonucleotide near the 5 end of a sense strand capable of mediating RNA interference (RNAi) against target nucleic acid sequences. The RNAi agents are useful in the treatment of diseases or conditions that respond to inhibition of gene expression or activity in a cell, tissue, or organism.本發明描述RNAi干擾劑,其適用於在多種應用中調節基因表現,包括用於治療、診斷、靶向驗證及基因組發現應用中。特定言之,本發明係關於經修飾之雙股寡核苷酸分子,其具有鈍端及至少一個接近有義股5端的核糖核苷酸,其能夠介導針對標靶核酸序列的RNA干擾(RNAi)。RNAi劑適用於治療對抑制細胞、組織或生物體中之基因表現或活性有反應的疾病或病狀。
