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

Autor: McNamee SM; Schepens Eye Research Institute, Massachusetts Eye and Ear, Department of Ophthalmology, Harvard Medical School, 20 Staniford Street, Boston, MA, 02114, USA., Akula M; Schepens Eye Research Institute, Massachusetts Eye and Ear, Department of Ophthalmology, Harvard Medical School, 20 Staniford Street, Boston, MA, 02114, USA., Love Z; Schepens Eye Research Institute, Massachusetts Eye and Ear, Department of Ophthalmology, Harvard Medical School, 20 Staniford Street, Boston, MA, 02114, USA., Nasraty N; Schepens Eye Research Institute, Massachusetts Eye and Ear, Department of Ophthalmology, Harvard Medical School, 20 Staniford Street, Boston, MA, 02114, USA., Nystuen K; University of Massachusetts Amherst, Amherst, MA, USA., Singh P; Ocugen, Inc., Malvern, PA, USA., Upadhyay AK; Ocugen, Inc., Malvern, PA, USA., DeAngelis MM; Department of Ophthalmology, Jacobs School of Medicine and Biomedical Sciences, University at Buffalo, Buffalo, NY, USA., Haider NB; Schepens Eye Research Institute, Massachusetts Eye and Ear, Department of Ophthalmology, Harvard Medical School, 20 Staniford Street, Boston, MA, 02114, USA. neena_haider@hms.harvard.edu.; Department of Cell Biology, Harvard Medical School, Boston, MA, 02138, USA. neena_haider@hms.harvard.edu.
Jazyk: angličtina
Zdroj: Scientific reports [Sci Rep] 2024 Jul 17; Vol. 14 (1), pp. 16490. Date of Electronic Publication: 2024 Jul 17.
DOI: 10.1038/s41598-024-67095-6
Abstrakt: 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 retinal degeneration in the rd7 mice. Our study revealed retinal morphology was significantly improved 6 months post for all doses in the early-stage treatment groups and for the low and mid doses in the intermediate stage treatment groups. Similarly, photoreceptor function was significantly improved in the early stage for all doses and intermediate stage low and mid dose groups 6 months post treatment. This study demonstrated efficacy in multiple doses of NR2E3 therapy.
(© 2024. The Author(s).)
Databáze: MEDLINE
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