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 - The Governor's Of The University Of Alberta

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 JP2021500064

Kind Code:

A

Abstract:

本発明は、ヒトジストロフィンプレｍＲＮＡのエクソン５１に結合してエクソンスキッピングを誘導する治療用アンチセンスオリゴヌクレオチド、ならびにＤＭＤの治療のためのそのコンジュゲートおよび組成物に関する。【選択図】図４

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Inventors:

Yokota Toshifumi
Yusuke Echigoya

Application Number:

JP2020524657A

Publication Date:

January 07, 2021

Filing Date:

July 20, 2018

Export Citation:

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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; C12N5/10; C12N15/88

Domestic Patent References:

JP2016521555A	2016-07-25
JP2013510561A	2013-03-28
JP2015522275A	2015-08-06
JP2011502118A	2011-01-20

Foreign References:

WO2011078797A2	2011-06-30
WO2003071872A1	2003-09-04

Other References:

脳と発達, vol. 42, JPN6022023364, 2010, pages 117 - 123, ISSN: 0004795079
NUCLEIC ACIDS RESEARCH, vol. 42, no. 12, JPN6023004520, 2014, pages 8174 - 8187, ISSN: 0004981361

Attorney, Agent or Firm:

Shinichiro Tanaka
▲吉▼田和彦
Hiroyuki Suda
Kazuo Yamazaki
Satsuki Ichikawa
Hironobu Hattori
Takamitsu Hoshino

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