Invention Grant
US08729025B2 Methods for altering MRNA splicing and treating familial dysautonomia by administering kinetin, benzyladenine, and tocotrienols
有权
通过施用激动素，苄基腺嘌呤和生育三烯酚来改变MRNA剪接和治疗家族性不自主的方法
- Patent Title: Methods for altering MRNA splicing and treating familial dysautonomia by administering kinetin, benzyladenine, and tocotrienols
- Patent Title (中): 通过施用激动素，苄基腺嘌呤和生育三烯酚来改变MRNA剪接和治疗家族性不自主的方法
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Application No.: US13601034Application Date: 2012-08-31
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Publication No.: US08729025B2Publication Date: 2014-05-20
- Inventor: Susan A. Slaugenhaupt , James F. Gusella
- Applicant: Susan A. Slaugenhaupt , James F. Gusella
- Applicant Address: US MA Boston
- Assignee: The General Hospital Corporation
- Current Assignee: The General Hospital Corporation
- Current Assignee Address: US MA Boston
- Agency: King & Spalding LLP
- Agent Kenneth H. Sonnenfeld
- Main IPC: A61K38/00
- IPC: A61K38/00 ; A61P3/04 ; A61P25/00 ; C07H21/02 ; C07H21/04
Abstract:
This invention relates to methods for altering the splicing of mRNA in cells. In particular, this invention also relates to methods for increasing the ratio of wild type to misspliced forms of mRNA and corresponding encoded proteins in cells possessing a mutant gene encoding either the i) misspliced mRNA corresponding to the mutant protein or ii) a component in the splicing machinery responsible for processing the misspliced mRNA. In addition, this invention relates to treating individuals having a disorder associated with a misspliced mRNA, such as Familial Dysautonomia or Neurofibromatosis 1, by administering to such an individual a cytokinin such as kinetin.
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