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.
本发明涉及改变细胞中mRNA剪接的方法。特别地,本发明还涉及增加野生型与错剪接形式的mRNA及相应编码的蛋白质在携带突变
基因编码i)错剪接的mRNA对应的突变蛋白或ii)负责处理错剪接mRNA的剪接机制组分的细胞中的比例的方法。此外,本发明还涉及通过向患有与错剪接mRNA相关的疾病,如家族性自主神经失调症或神经纤维瘤病1的个体,给予
细胞分裂素如
激动素治疗。