This subproject is one of many research subprojects utilizing theresources provided by a Center grant funded by NIH/NCRR. The subproject andinvestigator (PI) may have received primary funding from another NIH source,and thus could be represented in other CRISP entries. The institution listed isfor the Center, which is not necessarily the institution for the investigator.A number of promising treatments are being developed to treat retinal diseases such as age-related macular degeneration and retinitis pigmentosa. These potential treatments include delivery of gene therapy vectors or stem cells that release neurotrophic factors, both of which have been shown to prevent or delay retinal degeneration in rodent models. However, a substantial gap exists in our knowledge of how to translate these therapies from small animal studies to the clinic.
The aim of this project was to bridge this gap by using non-human primates (NHPs) to address the following aims: 1) to optimize the surgical techniques for subretinal gene and cell therapy delivery to the NHP eye and 2) conduct an initial test of the safety and biodistribution of these therapies. Non-human primates provide the essential stepping-stone from small animal studies to the treatment of humans with cell- and gene-based therapies. The rhesus macaque eye closely resembles the human eye in almost all respects. Most importantly, the macaque retina has a macula, a feature seen only in monkeys, apes and man. Furthermore, we have identified age-related drusen and pigmentary changes in macaque monkeys that closely mimic early to intermediate human AMD. Thus, these animals provide an ideal model for evaluation of efficacy of gene- and cell-based therapies for retinal disease.
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