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COMPOSITIONS FOR TREATING FAMILIAL DYSAUTONOMIA BY ALTERING MRNA SPLICING
专利权人:
THE GENERAL HOSPITAL CORPORATION
发明人:
SLAUGENHAUPT, SUSAN A.,GUSELLA, JAMES F.
申请号:
PL04794054
公开号:
PL1673439T3
申请日:
2004.10.01
申请国别(地区):
PL
年份:
2018
代理人:
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.
来源网站:
中国工程科技知识中心
来源网址:
http://www.ckcest.cn/home/
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