Department of Ophthalmology, Icahn School of Medicine at Mount Sinai, New York, NY 10029, USA; Department of Optometry and Vision Science, The University of Alabama at Birmingham, Birmingham, AL 35233, USA.
Department of Ophthalmology, Icahn School of Medicine at Mount Sinai, New York, NY 10029, USA; Department of Neuroscience, Icahn School of Medicine at Mount Sinai, New York, NY 10029, USA.
Vision Res. 2023 May;206:108196. doi: 10.1016/j.visres.2023.108196. Epub 2023 Feb 20.
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.
青光眼是一组疾病，通常以视神经变性为特征，是全球致盲的主要原因之一。虽然没有治愈青光眼的方法，但降低眼内压是一种已被认可的治疗方法，可以延迟大多数患者的视神经变性和视网膜神经节细胞（RGC）死亡。最近的临床试验评估了基因治疗载体治疗遗传性视网膜退行性疾病（IRDs）的安全性和疗效，结果令人鼓舞，为治疗其他视网膜疾病带来了热情。虽然尚无基因治疗神经保护治疗青光眼的成功临床试验报告，并且只有少数研究评估了基因治疗载体治疗莱伯遗传性视神经病变（LHON）的疗效，但神经保护治疗青光眼和其他影响 RGC 的疾病的潜力仍然得到广泛认可。在这里，我们回顾了最近的进展，并介绍了目前使用腺相关病毒（AAV）为载体的基因治疗靶向 RGC 治疗青光眼的局限性。
