Patent Portfolio

Composition for prevention or treatment of spinocerebellar ataxia type 36

Ref. No. AJ187
Appl. No.
(filing date)
PCT/JP2018/019082(2018/5/17)
Pub. No.
(Pub. date)
WO2018/212271(2018/11/22)
Priority claim No.
(Priority date)

JP2017-099374 (2017/05/18)

Patent No.

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Inventor INOUE, Haruhisa, etc.
Assignee Kyoto University
Title of Invention Composition for prevention or treatment of spinocerebellar ataxia type 36
Abstract

The present invention provides an antisense oligonucleotide (ASO) that effectively inhibits, without inducing cleavage of NOP56 pre-mRNA, the RNA foci that are spontaneously produced in neurons having SCA36 mutation. The oligonucleotide is complementary to pre-mRNA from the NOP56 gene and has a sequence in which a complementary sequence to a repeat unit within intron 1 of NOP56 pre-mRNA repeats at least one time, and is characterized in that the structure formed by hybridization to the pre-mRNA is RNaseH resistant.

Comments

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Updated date 2019/6/13
Information
(External link)
https://www.j-platpat.inpit.go.jp/c1800/PU/WO-A-2018-212271/884B322F4292CD4448D1FBC02622C839E61F93512233B8846B1EBA54F01BBCDA/50/en