The invention provides compositions comprising a TERT-RMRP or TERT-RNA complex and methods of treating subjects with genetic diseases in which gene silencing is either increased by administering the compositions of the invention or decreased by administering an inhibitor of the RNA-dependent RNA polymerase (RdRP) activity of these compositions. Moreover, the invention provides methods of screening for agonists and antagonists of RdRP activity and TERT-RMRP complex formation. Finally, the invention provides a method of identifying a RNA molecule that forms a complex with a TERT polypeptide and has RdRP activity.
本发明提供了包括TERT-RMRP或TERT-RNA复合物的组合物,并提供了使用该发明的组合物增加
基因沉默或通过给予RNA依赖性RNA聚合酶(RdRP)活性
抑制剂来减少
基因沉默的治疗遗传疾病的方法。此外,本发明还提供了筛选RdRP活性和TERT-RMRP复合物形成的激动剂和拮抗剂的方法。最后,本发明提供了一种识别形成与TERT
多肽复合物并具有RdRP活性的RNA分子的方法。