TRANSFER & EXPRESSION OF THE GFP REPORTER GENE FOLLOWING SINGLE SUBRETINAL

转移

• 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.

该副本是利用众多研究子项目之一 由NIH/NCRR资助的中心赠款提供的资源。子弹和 调查员（PI）可能已经从其他NIH来源获得了主要资金， 因此可以在其他清晰的条目中代表。列出的机构是 对于中心，这不一定是调查员的机构。 基因治疗对视网膜疾病的成功取决于发现最佳病毒载体以最小的副作用转导治疗基因。 腺相关病毒（AAV）载体已被证明是安全有效的，现在正在人类临床试验中使用。 但是，AAV通常只能携带相对较小的基因，因此需要其他病毒载体。 这项研究的目的是评估GFP记者基因构建体的转移安全性和效率，此前单次下递送的新基因治疗载体的单一递送剂量，并将结果与​​携带同一GFP报告基因的AAV Serotype-5载体进行比较。 将两个向量的注射均能耐受，没有副作用或明显的免疫反应。 使用两个载体，GFP基因在注射区域表达，并且没有视网膜结构的破坏。