METHODS FOR ALTERING MRNA SPLICING AND TREATING FAMILIAL DYSAUTONOMIA AND OTHER MECHANISTICALLY RELATED DISORDERS

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 ratioof wild type to misspliced forms of mRNA and corresponding encoded proteins incells possessing a mutant gene encoding either the i) misspliced mRNAcorresponding to the mutant protein or ii) a component in the splicingmachinery responsible for processing the misspliced mRNA. In addition, thisinvention relates to treating individuals having a disorder associated with amisspliced mRNA, such as Familial Dysautonomia or Neurofibromatosis 1, byadministering to such an individual a cytokinin such as kinetin.