TRANSFER & EXPRESSION OF THE GFP REPORTER GENE FOLLOWING SINGLE SUBRETINAL

这个子项目是许多研究子项目中利用 资源由NIH/NCRR资助的中心拨款提供。子项目和 调查员(PI)可能从NIH的另一个来源获得了主要资金， 并因此可以在其他清晰的条目中表示。列出的机构是 该中心不一定是调查人员的机构。 视网膜疾病基因治疗的成功依赖于找到最佳的病毒载体来转导副作用最小的治疗基因。腺相关病毒(AAV)载体已被证明是安全有效的，目前已在人体临床试验中使用。然而，AAV通常只能携带相对较小的基因，因此需要其他病毒载体。本研究的目的是评价单次视网膜下注射新型基因治疗载体后将GFP报告基因结构转移到恒河猴视网膜的安全性和有效性，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。注射两种载体均可耐受，无副作用或明显的免疫反应。在两种载体中，GFP基因都在注射区域表达，并且没有破坏视网膜的构筑。

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.