{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"submitter":["Fang F"],"funding":["NEI NIH HHS","National Eye Institute","National Natural Science Foundation of China"],"pagination":["1421-1431"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC9077370"],"repository":["biostudies-literature"],"omics_type":["Unknown"],"volume":["30(4)"],"pubmed_abstract":["The lack of neuroprotective treatments for retinal ganglion cells (RGCs) and optic nerve (ON) is a central challenge for glaucoma management. Emerging evidence suggests that redox factor NAD<sup>+</sup> decline is a hallmark of aging and neurodegenerative diseases. Supplementation with NAD<sup>+</sup> precursors and overexpression of NMNAT1, the key enzyme in the NAD<sup>+</sup> biosynthetic process, have significant neuroprotective effects. We first profile the translatomes of RGCs in naive mice and mice with silicone oil-induced ocular hypertension (SOHU)/glaucoma by RiboTag mRNA sequencing. Intriguingly, only NMNAT2, but not NMNAT1 or NMNAT3, is significantly decreased in SOHU glaucomatous RGCs, which we confirm by in situ hybridization. We next demonstrate that AAV2 intravitreal inject"],"journal":["Molecular therapy : the journal of the American Society of Gene Therapy"],"pubmed_title":["NMNAT2 is downregulated in glaucomatous RGCs, and RGC-specific gene therapy rescues neurodegeneration and visual function."],"pmcid":["PMC9077370"],"funding_grant_id":["R01 EY024932","R01 EY028106","R01 EY032159","R21 EY031063","R01 EY025295","R01 EY023295","P30 EY026877"],"pubmed_authors":["Zhuang P","Liu L","Hu Y","Feng X","Fang F","Huang H","Jiang H","Liu P","Li L","Sun Y","Chen W","Liu D","Ye J"],"additional_accession":[]},"is_claimable":false,"name":"NMNAT2 is downregulated in glaucomatous RGCs, and RGC-specific gene therapy rescues neurodegeneration and visual function.","description":"The lack of neuroprotective treatments for retinal ganglion cells (RGCs) and optic nerve (ON) is a central challenge for glaucoma management. Emerging evidence suggests that redox factor NAD<sup>+</sup> decline is a hallmark of aging and neurodegenerative diseases. Supplementation with NAD<sup>+</sup> precursors and overexpression of NMNAT1, the key enzyme in the NAD<sup>+</sup> biosynthetic process, have significant neuroprotective effects. We first profile the translatomes of RGCs in naive mice and mice with silicone oil-induced ocular hypertension (SOHU)/glaucoma by RiboTag mRNA sequencing. Intriguingly, only NMNAT2, but not NMNAT1 or NMNAT3, is significantly decreased in SOHU glaucomatous RGCs, which we confirm by in situ hybridization. We next demonstrate that AAV2 intravitreal inject","dates":{"release":"2022-01-01T00:00:00Z","publication":"2022 Apr","modification":"2026-05-09T22:16:13.647Z","creation":"2025-04-04T20:20:30.238Z"},"accession":"S-EPMC9077370","cross_references":{"pubmed":["35114390"],"doi":["10.1016/j.ymthe.2022.01.035"]}}