KONSTANTINOVA, PAVLINA STEFANOVA,MINIARIKOVA, JANA
申请号：
CA2971920
公开号：
CA2971920A1
申请日:
2015.12.23
申请国别(地区):
CA
年份:
2016
代理人:
摘要：
The present invention provides for a double stranded RNA comprising a first RNA sequence and a second RNA sequence wherein the first and second RNA sequence are substantially complementary, wherein the first RNA sequence has a sequence length of at least 19 nucleotides and is substantially complementary to SEQ ID NO. 1. Said double stranded RNA is for use in inducing RNAi against Huntingtin exon 1 sequences. The double stranded RNA of to the invention was capable of reducing neuronal cell death and huntingtin aggregates in an animal model.
