青光眼
神经保护
医学
视神经
遗传增强
视神经病变
视网膜神经节细胞
腺相关病毒
神经科学
视网膜
眼科
眼压
生物
载体(分子生物学)
药理学
基因
遗传学
重组DNA
作者
Christopher Starr,Bo Chen
标识
DOI:10.1016/j.visres.2023.108196
摘要
Glaucoma is a group of diseases typically characterized by the degeneration of the optic nerve and is one of the world's leading causes of blindness. Although there is no cure for glaucoma, reducing intraocular pressure is an approved treatment to delay optic nerve degeneration and retinal ganglion cell (RGC) death in most patients. Recent clinical trials have evaluated the safety and efficacy of gene therapy vectors for the treatment of inherited retinal degenerations (IRDs), and the results are promising, generating enthusiasm for the treatment of other retinal diseases. While there have been no reports on successful clinical trials for gene therapy-based neuroprotective treatment of glaucoma, and only a few studies assessing the efficacy of gene therapy vectors for the treatment of Leber hereditary optic neuropathy (LHON), the potential for neuroprotective treatment of glaucoma and other diseases affecting RGCs is still widely recognized. Here, we review recent progress and cover current limitations pertaining to targeting RGCs with adeno-associated virus-based gene therapy for the treatment of glaucoma.
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