PRIMATE RETINAL CELLS TREATED WITH GENE THERAPY TECHNIQUES

这个子项目是许多利用资源的研究子项目之一 由NIH/NCRR资助的中心拨款提供。子项目的主要支持 而子项目的主要调查员可能是由其他来源提供的， 包括其它NIH来源。 列出的子项目总成本可能 代表子项目使用的中心基础设施的估计数量， 而不是由NCRR赠款提供给子项目或子项目工作人员的直接资金。 在其他动物模型中的实验证据表明，在成人中使用基因治疗可以恢复视网膜功能。然而，基因治疗后对正常视网膜生理的影响尚未在细胞水平上研究。这是一个重要的问题，对于确定眼部基因治疗是否最终可用于治疗人类视力异常和疾病至关重要。我们将测试的模型系统是通过病毒载体掺入新的遗传物质后的非人灵长类动物视网膜生理学。在视网膜细胞中表达绿色荧光基因后，将在体外评估生理学和形态学。将对启动子基因进行修饰，以便潜在地靶向特定的视网膜细胞亚群。

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. Primary support for the subproject and the subproject's principal investigator may have been provided by other sources, including other NIH sources. The Total Cost listed for the subproject likely represents the estimated amount of Center infrastructure utilized by the subproject, not direct funding provided by the NCRR grant to the subproject or subproject staff. Experimental evidence in other animal models has demonstrated that retinal function can be restored using gene therapy in adults. However, the effects on normal retinal physiology following gene therapy has not been investigated at the cellular level. This is an important question that is central to determining whether ocular gene therapy can ultimately be used to treat human vision anomalies and disease. The model system we will test is non-human primate retinal physiology following incorporation of new genetic material via viral vector. Following expression of a gene for green fluorescence in retinal cells, physiology and morphology will be assessed in vitro. Modifications to the promoter genes will be made so as to potentially target specific retinal cell subpopulations.