发明申请
US20120329816A1 Methods for Altering MRNA Splicing and Treating Familial Dysautonomia and Other Mechanistically Related Disorders
有权
改变MRNA切割和治疗家族性自主神经病和其他机械相关性疾病的方法
- 专利标题： Methods for Altering MRNA Splicing and Treating Familial Dysautonomia and Other Mechanistically Related Disorders
- 专利标题（中）： 改变MRNA切割和治疗家族性自主神经病和其他机械相关性疾病的方法
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申请号： US13601034申请日： 2012-08-31
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公开(公告)号： US20120329816A1公开(公告)日： 2012-12-27
- 发明人: Susan A. Slaugenhaupt , James F. Gusella
- 申请人： Susan A. Slaugenhaupt , James F. Gusella
- 申请人地址： US MA Boston
- 专利权人： The General Hospital Corporation
- 当前专利权人： The General Hospital Corporation
- 当前专利权人地址： US MA Boston
- 主分类号： A61K31/52
- IPC分类号： A61K31/52 ; A61P25/00 ; A61P25/28
摘要：
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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