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Preclinical dose response study shows NR2E3 can attenuate retinal degeneration in the retinitis pigmentosa mouse model RhoP23H+/.
McNamee, Shannon M; Chan, Natalie P; Akula, Monica; Avola, Marielle O; Whalen, Maiya; Nystuen, Kaden; Singh, Pushpendra; Upadhyay, Arun K; DeAngelis, Margaret M; Haider, Neena B.
Afiliación
  • McNamee SM; Schepens Eye Research Institute, Massachusetts Eye and Ear, Department of Ophthalmology, Harvard Medical School, Boston, MA, USA.
  • Chan NP; Schepens Eye Research Institute, Massachusetts Eye and Ear, Department of Ophthalmology, Harvard Medical School, Boston, MA, USA.
  • Akula M; Schepens Eye Research Institute, Massachusetts Eye and Ear, Department of Ophthalmology, Harvard Medical School, Boston, MA, USA.
  • Avola MO; Schepens Eye Research Institute, Massachusetts Eye and Ear, Department of Ophthalmology, Harvard Medical School, Boston, MA, USA.
  • Whalen M; Schepens Eye Research Institute, Massachusetts Eye and Ear, Department of Ophthalmology, Harvard Medical School, Boston, MA, 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 & Biomedical Sciences, University at Buffalo, Buffalo, NY, USA.
  • Haider NB; Schepens Eye Research Institute, Massachusetts Eye and Ear, Department of Ophthalmology, Harvard Medical School, Boston, MA, USA. neena_haider@hms.harvard.edu.
Gene Ther ; 31(5-6): 255-262, 2024 05.
Article en En | MEDLINE | ID: mdl-38273095
ABSTRACT
Retinitis pigmentosa (RP) is a heterogeneous disease and the main cause of vision loss within the group of inherited retinal diseases (IRDs). IRDs are a group of rare disorders caused by mutations in one or more of over 280 genes which ultimately result in blindness. Modifier genes play a key role in modulating disease phenotypes, and mutations in them can affect disease outcomes, rate of progression, and severity. Our previous studies have demonstrated that the nuclear hormone receptor 2 family e, member 3 (Nr2e3) gene reduced disease progression and loss of photoreceptor cell layers in RhoP23H-/- mice. This follow up, pharmacology study evaluates a longitudinal NR2E3 dose response in the clinically relevant heterozygous RhoP23H mouse. Reduced retinal degeneration and improved retinal morphology was observed 6 months following treatment evaluating three different NR2E3 doses. Histological and immunohistochemical analysis revealed regions of photoreceptor rescue in the treated retinas of RhoP23H+/- mice. Functional assessment by electroretinogram (ERG) showed attenuated photoreceptor degeneration with all doses. This study demonstrates the effectiveness of different doses of NR2E3 at reducing retinal degeneration and informs dose selection for clinical trials of RhoP23H-associated RP.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Degeneración Retiniana / Retinitis Pigmentosa / Modelos Animales de Enfermedad / Receptores Nucleares Huérfanos Límite: Animals Idioma: En Revista: Gene Ther Asunto de la revista: GENETICA MEDICA / TERAPEUTICA Año: 2024 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Reino Unido
Texto completo
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Degeneración Retiniana / Retinitis Pigmentosa / Modelos Animales de Enfermedad / Receptores Nucleares Huérfanos Límite: Animals Idioma: En Revista: Gene Ther Asunto de la revista: GENETICA MEDICA / TERAPEUTICA Año: 2024 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Reino Unido