EXTERNAL GUIDE SEQUENCE (EGS) AND AIDS THERAPY

描述：(改编自申请者摘要)本提案基于 发现被RNaseP识别的结构可以被引入 任何外源RNA(例如，mRNAs)通过与小分子的 寡核苷酸导致靶基因的切割。因此，这样的 小RNA或外部引导序列可用于发展 治疗人类遗传性疾病和传染性疾病的新方法 疾病。拟议的目标是：(I)通过以下方式优化EGS的效率 确定切割和靶向效率的最佳结构和 (2)开发以EGS为基础的治疗艾滋病毒的方法 通过靶向CCR5mRNA感染。

DESCRIPTION: (Adapted From Applicant's Abstract) This proposal is based on the finding that structures that are recognized by RNAse P can be introduced into any foreign RNA (e. g. mRNA) via hybridization to a small oligoribonucleotide resulting in cleavage of the targeted mRNA. Thus, such small RNAs or external guide sequences can be used in the development of novel approaches toward treatment of human genetic disorders and infectious diseases. The proposed goals are: (i) to optimize the EGS efficiency via determining the optimal structure for cleavage and targeting efficiency and (ii) to develop an EGS-based therapeutic approach to the treatment of HIV infection by targeting CCR5 mRNA.