TRANSFER & EXPRESSION OF THE GFP REPORTER GENE FOLLOWING SINGLE SUBRETINAL

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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.