EXTERNAL GUIDE SEQUENCE (EGS) AND AIDS THERAPY

描述：（改编自申请人的摘要）本提案基于 这一发现，即可以引入被RNA酶P识别的结构， 任何外源RNA（e. G. mRNA）通过与小的 在一些实施方案中，所述寡核糖核苷酸可导致靶向mRNA的切割。 因此， 小RNA或外部指导序列可用于开发 治疗人类遗传性疾病和传染性疾病的新方法 疾病 提出的目标是：（一）优化EGS效率， 确定切割和靶向效率的最佳结构， (ii)开发一种基于EGS的艾滋病治疗方法， 通过靶向CCR5 mRNA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.