He Meihui, Rong Rong, Ji Dan, Xia Xiaobo
Eye Center of Xiangya Hospital, Central South University, Changsha, China.
Hunan Key Laboratory of Ophthalmology, Changsha, China.
Front Cell Dev Biol. 2022 May 16;10:879957. doi: 10.3389/fcell.2022.879957. eCollection 2022.
Glaucoma is a group of optic neuropathies featured by degeneration of retinal ganglion cells and loss of their axons in the optic nerve. The only currently approved therapies focus on lowering intraocular pressure with medication and surgery. Over the previous few decades, technological advances and research progress regarding pathogenesis has brought glaucomatous gene therapy to the forefront. In this review, we discuss the three current genome editing methods and potential disease mechanisms of glaucoma. We further summarize different genome editing strategies that are being developed to target a number of glaucoma-related genes and pathways from four aspects including strategies to lower intraocular pressure, neuroprotection, RGC and optic nerve neuro-regeneration, and other strategies. In summary, genome therapy is a promising therapy for treating patients with glaucoma and has great potential to be widely applied in clinical practice.
青光眼是一组以视网膜神经节细胞变性及其视神经轴突丧失为特征的视神经病变。目前唯一获批的治疗方法是通过药物和手术降低眼压。在过去几十年中，技术进步和发病机制研究进展使青光眼基因治疗成为前沿领域。在本综述中，我们讨论了当前三种基因组编辑方法以及青光眼的潜在疾病机制。我们进一步从降低眼压策略、神经保护、视网膜神经节细胞和视神经神经再生以及其他策略四个方面总结了正在开发的针对多个青光眼相关基因和通路的不同基因组编辑策略。总之，基因治疗是治疗青光眼患者的一种有前景的疗法，具有在临床实践中广泛应用的巨大潜力。
