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