Title:
Regulation of myotonic dystrophy protein kinase (DMPK) expression
Document Type and Number:
Japanese Patent JP6343308
Kind Code:
B2
Abstract:
Provided herein are methods, compounds, and compositions for reducing expression of a DMPK mRNA and protein in an animal. Also provided herein are methods, compounds, and compositions for preferentially reducing CUGexp DMPK RNA, reducing myotonia or reducing spliceopathy in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate type 1 myotonic dystrophy, or a symptom thereof.
Inventors:
Sea Frank Bennett
Susan M Flair
Robert A. McLeod
Sanjay Kay Pandy
Charles A. Thornton
Thurman Wheeler
Suong H Chu Young
Andrew Leger
Bruce M Wentworth
Susan M Flair
Robert A. McLeod
Sanjay Kay Pandy
Charles A. Thornton
Thurman Wheeler
Suong H Chu Young
Andrew Leger
Bruce M Wentworth
Application Number:
JP2016147984A
Publication Date:
June 13, 2018
Filing Date:
July 28, 2016
Export Citation:
Assignee:
Ionis Pharmaceuticals,Inc.
International Classes:
C12N15/113; A61K31/7088; A61K45/00; A61K48/00; A61P21/00; A61P21/04; C12N15/09
Domestic Patent References:
| JP2010500023A |
Foreign References:
| WO2004093783A1 | ||||
| US20100016215 |
Other References:
RNA & Oligonucleotide therapeutics, 2010 April, p.35
Science, 2009, vol.325, no.5938, p.336-339
Science, 2009, vol.325, no.5938, p.336-339
Attorney, Agent or Firm:
Shinjiro Ono
Yasushi Kobayashi
Shigeo Takeuchi
Osamu Yamamoto
Yasushi Kobayashi
Shigeo Takeuchi
Osamu Yamamoto
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