Title:
筋緊張性ジストロフィーのためのタンパク質置換治療に関する組成物および方法
Document Type and Number:
Japanese Patent JP5926475
Kind Code:
B2
Abstract:
Disclosed are antisense oligonucleotides for use in treating myotonic dystrophy (DM1 or DM2). The oligonucleotides either inhibit the interaction of poly(CUG) exp or poly(CCUG) exp RNA with muscleblind proteins (MBNL1) or may improve spliceopathy in relation to chloride ion channel ClC-1.
Inventors:
Thornton, Charles A.
Wheeler, Thurman
Sobezak, Krushishtov
Robert Osborne
Mirror, jill
Swanson, Maurice S.
Wheeler, Thurman
Sobezak, Krushishtov
Robert Osborne
Mirror, jill
Swanson, Maurice S.
Application Number:
JP2009529258A
Publication Date:
May 25, 2016
Filing Date:
September 21, 2007
Export Citation:
Assignee:
UNIVERSITY OF ROCHESTER
University of Florida Research Foundation, Incorporated
University of Florida Research Foundation, Incorporated
International Classes:
C12N15/09; A61K31/7036; A61K31/7088; A61K45/00; A61K48/00; A61P21/04; A61P43/00
Domestic Patent References:
| JP2003518948A |
Foreign References:
| WO2005010532A1 |
Other References:
PNAS、2006.08.01、Vol.103、p.11748-11753
Attorney, Agent or Firm:
Hidesaku Yamamoto
Natsuki Morishita
Natsuki Morishita