BUTLER, JIM,BETTENCOURT, BRIAN,RAJEEV, KALLANTHOTTATHIL G.,MAIER, MARTIN,CHARISSE, KLAUS
申请号：
TW103117873
公开号：
TW201536916A
申请日:
2014.05.22
申请国别(地区):
TW
年份:
2015
代理人:
摘要：
The invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the TMPRSS6 gene, and methods of using such RNAi agents to inhibit expression of TMPRSS6 and methods of treating subjects having a TMPRSS6 associated disorder, e.g., an iron overload associated disorder, such as [beta]-thalassemia or hemochromatosis.本發明係關於以TMPRSS6基因為靶向之RNAi劑，如雙股RNAi劑，及使用此等RNAi劑抑制TMPRSS6之表現的方法，及治療具有TMPRSS6相關病症，如鐵過載相關病症，如β-地中海貧血或血色素沉著症之受驗者的方法。
