دورية أكاديمية
Small-Molecule-Directed Endogenous Regeneration of Visual Function in a Mammalian Retinal Degeneration Model
| العنوان: | Small-Molecule-Directed Endogenous Regeneration of Visual Function in a Mammalian Retinal Degeneration Model |
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| المؤلفون: | Daphna Mokady, Jason Charish, Patrick Barretto-Burns, Kenneth N. Grisé, Brenda L. K. Coles, Susanne Raab, Arturo Ortin-Martinez, Alex Müller, Bernhard Fasching, Payal Jain, Micha Drukker, Derek van der Kooy, Matthias Steger |
| المصدر: | International Journal of Molecular Sciences, Vol 25, Iss 3, p 1521 (2024) |
| بيانات النشر: | MDPI AG, 2024. |
| سنة النشر: | 2024 |
| المجموعة: | LCC:Biology (General) LCC:Chemistry |
| مصطلحات موضوعية: | small molecule, regenerative medicine, inherited retinal diseases, retina, photoreceptors, functional recovery, Biology (General), QH301-705.5, Chemistry, QD1-999 |
| الوصف: | Degenerative retinal diseases associated with photoreceptor loss are a leading cause of visual impairment worldwide, with limited treatment options. Phenotypic profiling coupled with medicinal chemistry were used to develop a small molecule with proliferative effects on retinal stem/progenitor cells, as assessed in vitro in a neurosphere assay and in vivo by measuring Msx1-positive ciliary body cell proliferation. The compound was identified as having kinase inhibitory activity and was subjected to cellular pathway analysis in non-retinal human primary cell systems. When tested in a disease-relevant murine model of adult retinal degeneration (MNU-induced retinal degeneration), we observed that four repeat intravitreal injections of the compound improved the thickness of the outer nuclear layer along with the regeneration of the visual function, as measured with ERG, visual acuity, and contrast sensitivity tests. This serves as a proof of concept for the use of a small molecule to promote endogenous regeneration in the eye. |
| نوع الوثيقة: | article |
| وصف الملف: | electronic resource |
| اللغة: | English |
| تدمد: | 1422-0067 1661-6596 |
| Relation: | https://www.mdpi.com/1422-0067/25/3/1521; https://doaj.org/toc/1661-6596; https://doaj.org/toc/1422-0067 |
| DOI: | 10.3390/ijms25031521 |
| URL الوصول: | https://doaj.org/article/52c1c22b770c4c6e9d3d737724110ca2 |
| رقم الأكسشن: | edsdoj.52c1c22b770c4c6e9d3d737724110ca2 |
| قاعدة البيانات: | Directory of Open Access Journals |
Full Text Finder | تدمد: | 14220067 16616596 |
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| DOI: | 10.3390/ijms25031521 |