Evaluating therapeutic potential of NR2E3 doses in the rd7 mouse model of retinal degeneration.

ABSTRACT: Retinitis Pigmentosa is a leading cause of severe vision loss. Retinitis Pigmentosa can present with a broad range of phenotypes impacted by disease age of onset, severity, and progression. This variation is influenced both by different gene mutations as well as unique variants within the same gene. Mutations in the nuclear hormone receptor 2 family e, member 3 are associated with several forms of retinal degeneration, including Retinitis Pigmentosa. In our previous studies we demonstrated that subretinal administration of one Nr2e3 dose attenuated retinal degeneration in rd7 mice for at least 3 months. Here we expand the studies to evaluate the efficacy and longitudinal impact of the NR2E3 therapeutic by examining three different doses administered at early or intermediate stages of retin

SUBMITTER: McNamee SM 

PROVIDER: S-EPMC11254931 | biostudies-literature | 2024 Jul

REPOSITORIES: biostudies-literature