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.

该子项目是利用该技术的众多研究子项目之一 资源由 NIH/NCRR 资助的中心拨款提供。子项目和 研究者 (PI) 可能已从 NIH 的另一个来源获得主要资金， 因此可以在其他 CRISP 条目中表示。列出的机构是 对于中心来说，它不一定是研究者的机构。 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转导治疗基因。 腺相关病毒（AAV）载体已被证明安全有效，目前已用于人体临床试验。 然而，AAV一般只能携带相对较小的基因，因此需要其他病毒载体。 本研究的目的是评估单次视网膜下递送剂量的新型基因治疗载体后 GFP 报告基因构建体转移至恒河猴视网膜的安全性和效率，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。 两种载体的注射均可耐受，没有副作用或显着的免疫反应。 对于这两种载体，GFP 基因都在注射区域表达，并且视网膜结构没有受到破坏。