Eye Program, Board of Governors Regenerative Medicine Institute and Department of Biomedical Sciences, Cedars- Sinai Medical Center, Los Angeles, CA, USA.
Sackler School of Medicine, New York State/American Program of Tel Aviv University, Tel Aviv, Israel.
Curr Gene Ther. 2022;22(2):104-131. doi: 10.2174/1566523221666210423084233.
This review provides comprehensive information about the advances in gene therapy in the anterior segment of the eye, including cornea, conjunctiva, lacrimal gland, and trabecular meshwork. We discuss gene delivery systems, including viral and non-viral vectors as well as gene editing techniques, mainly CRISPR-Cas9, and epigenetic treatments, including antisense and siRNA therapeutics. We also provide a detailed analysis of various anterior segment diseases where gene therapy has been tested with corresponding outcomes. Disease conditions include corneal and conjunctival fibrosis and scarring, corneal epithelial wound healing, corneal graft survival, corneal neovascularization, genetic corneal dystrophies, herpetic keratitis, glaucoma, dry eye disease, and other ocular surface diseases. Although most of the analyzed results on the use and validity of gene therapy at the ocular surface have been obtained in vitro or using animal models, we also discuss the available human studies. Gene therapy approaches are currently considered very promising as emerging future treatments of various diseases, and this field is rapidly expanding.
这篇综述提供了关于眼部前段（包括角膜、结膜、泪腺和小梁网）基因治疗进展的全面信息。我们讨论了基因传递系统，包括病毒和非病毒载体以及基因编辑技术，主要是 CRISPR-Cas9，和表观遗传治疗，包括反义寡核苷酸和 siRNA 治疗。我们还详细分析了各种前段疾病，其中基因治疗已在相应的结果中进行了测试。疾病情况包括角膜和结膜纤维化和瘢痕形成、角膜上皮伤口愈合、角膜移植物存活、角膜新生血管形成、遗传性角膜营养不良、疱疹性角膜炎、青光眼、干眼症和其他眼表面疾病。尽管在眼表面使用基因治疗的大多数分析结果都是在体外或使用动物模型获得的，但我们也讨论了现有的人体研究。基因治疗方法目前被认为是非常有前途的新兴未来治疗各种疾病的方法，并且该领域正在迅速发展。
