Invention Grant
- Patent Title: Compositions and methods for modulation of SMN2 splicing
- Patent Title (中): SMN2拼接调制的组成和方法
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Application No.: US13720474Application Date: 2012-12-19
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Publication No.: US08946183B2Publication Date: 2015-02-03
- Inventor: Brenda F. Baker , Adrian R. Krainer , Yimin Hua
- Applicant: Isis Pharmaceuticals, Inc. , Cold Spring Harbor Laboratory
- Applicant Address: US CA Carlsbad
- Assignee: Isis Pharmaceuticals, Inc.
- Current Assignee: Isis Pharmaceuticals, Inc.
- Current Assignee Address: US CA Carlsbad
- Agency: Knobbe Martens LLP
- Main IPC: C07H21/02
- IPC: C07H21/02 ; C07H21/04 ; A61K31/70 ; C12N15/113 ; C12N15/11
Abstract:
Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a cell, tissue or animal. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy.
Public/Granted literature
- US20130109091A1 COMPOSITIONS AND METHODS FOR MODULATION OF SMN2 SPLICING Public/Granted day:2013-05-02
Information query
