TRANSFER & EXPRESSION OF THE GFP REPORTER GENE FOLLOWING SINGLE SUBRETINAL

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• 批准号: 8173313
• 负责人: MARTHA NEURINGER
• 金额: $ 4.76万
• 依托单位: OREGON HEALTH & SCIENCE UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2010
• 资助国家: 美国
• 起止时间: 2010-05-01 至 2011-04-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8173313
• 关键词: Adverse effects; Architecture; Area; Clinical Trials; Computer Retrieval of Information on Scientific Projects Database; Dose; Funding; Gene Transduction Agent; Genes; Grant; Human; Immune response; Injection of therapeutic agent; Institution; Macaca mulatta; Reporter Genes; Research; Research Personnel; Resources; Retina; Retinal; Retinal Diseases; Safety; Serotyping; Source; United States National Institutes of Health; Viral; Viral Vector; adeno-associated viral vector; gene therapy; novel; success; therapeutic gene; vector

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. The success of gene therapy for retinal disorders depends on finding the optimal viral vectors to transduce the therapeutic gene with minimal side effects. Adeno-associated viral (AAV) vectors have been demonstrated to be safe and effective, and are now in use in human clinical trials. However, AAV is generally only capable of carrying relatively small genes, and therefore there is a need for other viral vectors. The object of this study was to assess safety and efficiency of transfer of the GFP reporter gene construct to the rhesus macaque retina following a single subretinally delivered dose of a novel gene therapy vector, and to compare the results with an AAV serotype-5 vector carrying the same GFP reporter gene. Injection of both vectors was tolerated without side effects or significant immune response. With both vectors, the GFP gene was expressed in the area of the injection, and there was no disruption of retinal architecture.

这个子项目是许多研究子项目中的一个 由NIH/NCRR资助的中心赠款提供的资源。子项目和 研究者（PI）可能从另一个NIH来源获得了主要资金， 因此可以在其他CRISP条目中表示。所列机构为 研究中心，而研究中心不一定是研究者所在的机构。 视网膜疾病基因治疗的成功取决于找到最佳的病毒载体，以最小的副作用来包裹治疗基因。 腺相关病毒（AAV）载体已被证明是安全和有效的，现在在人体临床试验中使用。 然而，AAV通常仅能够携带相对小的基因，因此需要其他病毒载体。 本研究的目的是评估GFP报告基因构建体转移到恒河猴视网膜后，单次视网膜下递送剂量的新型基因治疗载体的安全性和效率，并将结果与携带相同GFP报告基因的AAV血清型-5载体进行比较。 两种载体的注射都是耐受的，没有副作用或显著的免疫应答。 使用两种载体，GFP基因在注射区域中表达，并且没有破坏视网膜结构。