Invention Grant
US08741572B1 Methods of screening for compounds for treating muscular dystrophy using mIGF1 mRNA translation regulation
失效
使用mIGF1 mRNA翻译调节法筛选化合物治疗肌营养不良的方法
- Patent Title: Methods of screening for compounds for treating muscular dystrophy using mIGF1 mRNA translation regulation
- Patent Title (中): 使用mIGF1 mRNA翻译调节法筛选化合物治疗肌营养不良的方法
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Application No.: US13646943Application Date: 2012-10-08
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Publication No.: US08741572B1Publication Date: 2014-06-03
- Inventor: Westley J. Friesen , Nikolai Naryshkin , Meenal Patel , Charles Romfo , Yuki Tomizawa , Ellen Welch , Jin Zhou
- Applicant: PTC Therapeutics, Inc.
- Applicant Address: US NJ South Plainfield
- Assignee: PTC Therapeutics, Inc.
- Current Assignee: PTC Therapeutics, Inc.
- Current Assignee Address: US NJ South Plainfield
- Agency: Jones Day
- Main IPC: C12Q1/68
- IPC: C12Q1/68 ; C12N15/82 ; C12N15/113 ; C07K14/65

Abstract:
The present invention provides compounds and assays for the identification and validation of compounds for use in the treatment of muscular mystrophy (MD), or a form thereof, in which said compounds increase the post-transcriptional expression of a target gene (i.e., mIGF1, ITGA7, or UTRN).
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