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標靶性配體
专利权人:
ARROWHEAD PHARMACEUTICALS; INC.
发明人:
LI, ZHEN,李䂦□,PEI, TAO,裴 涛,裴 濤,GLEBOCKA, AGNIESKA,葛利波卡 安格尼斯卡,LAWLER, MICHAEL,罗勒 麦可,羅勒 麥可,FLEITZ, FRED,弗立兹 佛瑞德,弗立茲 佛瑞德,ALTENHOFER, ERICH,欧廷霍夫 艾瑞奇,歐廷霍夫 艾瑞奇,KUMAR, PANKAJ,库玛 潘卡,庫瑪 潘卡,裴 涛,裴 濤,葛利波卡 安格尼斯卡,罗勒 麦可,羅勒 麥可,弗立兹 佛瑞德,弗立茲 佛瑞德,欧廷霍夫 艾瑞奇,歐廷霍夫 艾瑞奇,库玛 潘卡,庫瑪 潘卡
申请号:
TW106107455
公开号:
TW201811375A
申请日:
2017.03.07
申请国别(地区):
TW
年份:
2018
代理人:
摘要:
Described are novel targeting ligands that may be linked to compounds, such therapeutic compounds that are useful in directing the compounds to the in vivo target. The targeting ligands disclosed herein can serve to target expression-inhibiting oligomeric compounds, such as RNAi agents, to liver cells to modulate gene expression. The targeting ligands disclosed herein, when conjugated to a therapeutic compound, may be used in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Compositions including the targeting ligands disclosed herein when linked to expression-inhibiting oligomeric compounds are capable of mediating expression of target nucleic acid sequences in liver cells, such as hepatocytes, which may be useful in the treatment of diseases or conditions that respond to inhibition of gene expression or activity in a cell, tissue, or organism.本文描述可連接至化合物(諸如治療化合物)之新穎標靶性配體,其可用於將該等化合物引導至活體內標靶。本文揭示之標靶性配體可用以將抑制表現之寡聚化合物(諸如RNAi劑)靶向肝臟細胞以調節基因表現。本文揭示之標靶性配體在結合至治療化合物時可用於各種應用中,包括用於治療、診斷、標靶確認及基因體發現應用中。包括本文揭示之標靶性配體之組合物在結合至抑制表現之寡聚化合物時可介導肝臟細胞(諸如肝細胞(hepatocytes))中標靶核酸序列之表現,可用於治療對於細胞、組織或生物體中基因表現或活性之抑制有反應之疾病或病症。
来源网站:
中国工程科技知识中心
来源网址:
http://www.ckcest.cn/home/

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