EXTERNAL GUIDE SEQUENCE (EGS) AND AIDS THERAPY

外部引导序列 (EGS) 和艾滋病治疗

• 批准号: 2429047
• 负责人: YAN YUAN
• 金额: $ 10.34万
• 依托单位: UNIVERSITY OF PENNSYLVANIA
• 依托单位国家: 美国
• 财政年份: 1997
• 资助国家: 美国
• 起止时间: 1997-08-01 至 2002-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/2429047
• 关键词: AIDS therapy; RNA; endoribonucleases; gene therapy; human immunodeficiency virus 1; nucleic acid structure; site directed mutagenesis; technology /technique development; tissue /cell culture; virus receptors

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.

描述：（改编自申请人的摘要）此提案基于 可以引入RNase P识别的结构的发现 通过与小的杂交进入任何外国RNA（例如mRNA） 寡核苷酸导致靶向mRNA裂解。 因此，这样的 小型RNA或外部指南序列可用于开发 治疗人遗传疾病和感染性的新方法 疾病。 提议的目标是：（i）通过 确定切割和靶向效率的最佳结构 （ii）开发一种基于EGS的治疗方法来治疗HIV 通过靶向CCR5 mRNA感染。