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.

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