Title:
ＲＮＡｉで誘発されるハンチンチン遺伝子抑制
Document Type and Number:
Japanese Patent JP6839094
Kind Code:
B2
Abstract:
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.
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Inventors:
Pavrina Stefanonova Constantinova
Yana Mini Alikova
Yana Mini Alikova
Application Number:
JP2017552234A
Publication Date:
March 03, 2021
Filing Date:
December 23, 2015
Export Citation:
Assignee:
Uniqua IPBee
International Classes:
C12N15/113; A61K31/713; A61K48/00; A61P25/14; C12N1/15; C12N1/19; C12N1/21; C12N5/10
Other References:
MOLECULAR THERAPY, 2005, Vol.12, pp.618-633
Molecular Therapy, 2008, Vol.16, pp.947-956
Molecular Therapy, 2008, Vol.16, pp.947-956
Attorney, Agent or Firm:
Yasuhiko Murayama
Shinya Mitsuhiro
Tatsuhiko Abe
Shinya Mitsuhiro
Tatsuhiko Abe