Title:
【発明の名称】スプライス領域アンチセンス組成物および方法
Document Type and Number:
Japanese Patent JP2003533986
Kind Code:
A
Abstract:
Antisense compositions targeted against an mRNA sequence coding for a selected protein, at a region having its 5′ end from 1 to about 25 base pairs downstream of a normal splice acceptor junction in the preprocessed mRNA, are disclosed. The antisense compound is RNase-inactive, and is preferably a phosphorodiamidate-linked morpholino oligonucleotide. Such targeting is effective to inhibit natural mRNA splice processing, produce splice variant mRNAs, and inhibit normal expression of the protein.
Inventors:
Ibersen, Patrick El.
Hughiak, Robert
Hughiak, Robert
Application Number:
JP2001580347A
Publication Date:
November 18, 2003
Filing Date:
May 04, 2001
Export Citation:
Assignee:
AVA I By Offeroma, Inc.
International Classes:
C12N15/09; A61K31/7105; A61K48/00; A61P5/06; A61P5/28; A61P9/10; A61P13/08; A61P17/00; A61P31/04; A61P31/12; A61P31/18; A61P31/20; A61P35/00; A61P35/02; A61P37/04; A61P43/00; C07F9/6558; C12N5/10; C12N15/113; A61K38/00; (IPC1-7): C12N15/09; A61K31/7105; A61K48/00; A61P5/06; A61P5/28; A61P9/10; A61P13/08; A61P17/00; A61P31/04; A61P31/12; A61P31/18; A61P31/20; A61P35/00; A61P35/02; A61P37/04; A61P43/00; C12N5/10
Domestic Patent References:
| JP2005504522A | 2005-02-17 |
Attorney, Agent or Firm:
Shusaku Yamamoto (2 outside)