Title:
ヒトジストロフィンプレｍＲＮＡのエクソン５１に結合するアンチセンスオリゴヌクレオチド
Document Type and Number:
Japanese Patent JP7365336
Kind Code:
B2
Abstract:
The present invention relates to a therapeutic antisense oligonucleotide which binds to exon 51 of the human dystrophin pre-mRNA to induce exon skipping, and conjugates and compositions thereof for the treatment of DMD.
Inventors:
Toshifumi Yokota
Yusuke Echigoya
Yusuke Echigoya
Application Number:
JP2020524657A
Publication Date:
October 19, 2023
Filing Date:
July 20, 2018
Export Citation:
Assignee:
The Governor's Of The University Of Alberta
International Classes:
C12N15/113; A61K31/711; A61K47/54; A61K47/56; A61K47/62; A61K47/69; A61K48/00; A61P21/00; A61P21/04; C07H21/04; C12N15/88
Domestic Patent References:
| JP2016521555A | ||||
| JP2013510561A | ||||
| JP2015522275A | ||||
| JP2011502118A |
Foreign References:
| WO2011078797A2 | ||||
| WO2003071872A1 |
Other References:
脳と発達,2010年,42,p. 117-123
Attorney, Agent or Firm:
Shinichiro Tanaka
Hiroyuki Suda
Kazuo Yamazaki
Satsuki Ichikawa
Hironobu Hattori
Nanae Matsuda
Takamitsu Hoshino
Hiroyuki Suda
Kazuo Yamazaki
Satsuki Ichikawa
Hironobu Hattori
Nanae Matsuda
Takamitsu Hoshino