发明公开
EP1673439A2 METHODS FOR ALTERING MRNA SPLICING AND TREATING FAMILIAL DYSAUTONOMIA AND OTHER MECHANISTICALLY RELATED DISORDERS
有权
ZUSAMMENSETZUNGEN ZUR BEHANDLUNG VONFAMILIÄRERDYSAUTONOMIE DURCHÄNDERUNGDES SPLEISSENS VON MRNA
- 专利标题： METHODS FOR ALTERING MRNA SPLICING AND TREATING FAMILIAL DYSAUTONOMIA AND OTHER MECHANISTICALLY RELATED DISORDERS
- 专利标题（中）： ZUSAMMENSETZUNGEN ZUR BEHANDLUNG VONFAMILIÄRERDYSAUTONOMIE DURCHÄNDERUNGDES SPLEISSENS VON MRNA
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申请号： EP04794054.9申请日： 2004-10-01
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公开(公告)号： EP1673439A2公开(公告)日： 2006-06-28
- 发明人: SLAUGENHAUPT, Susan, A. , GUSELLA, James, F.
- 申请人： THE GENERAL HOSPITAL CORPORATION
- 申请人地址： 55 Fruit Street Boston, MA 02114 US
- 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： 55 Fruit Street Boston, MA 02114 US
- 代理机构： Williams, Gareth Owen
- 优先权： US508465P 20031003; US536287P 20040113
- 国际公布： WO2005033290 20050414
- 主分类号： C12N1/00
- IPC分类号： C12N1/00
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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