Krajewska Julia B, Waszczykowska Arleta
Department of Ophthalmology, Norbert Barlicki Memorial Teaching Hospital No. 1, Lodz, Poland.
Department of Ophthalmology, Medical University of Lodz, Kopcinskiego 22, 90-153, Lodz, Poland.
J Appl Genet. 2025 Jun 5. doi: 10.1007/s13353-025-00973-5.
Gene therapies have recently emerged as promising strategies for treatment of previously incurable or poorly managed diseases. These hopes are particularly high in ophthalmology, as the eye is considered exceptionally suited for gene therapy. Expansion of gene therapy products may change the clinical course of treatment and give new chances to many patients. In this review, we address treatment possibilities and delivery methods as well as challenges and uncertainties related to gene therapy. We present inherited and acquired diseases which are subject to studies within this area, summarize current trends in ocular gene therapy, and indicate the future directions.
基因疗法最近已成为治疗先前无法治愈或治疗效果不佳疾病的有前景的策略。在眼科领域，人们对基因疗法的期望尤其高，因为眼睛被认为特别适合基因治疗。基因治疗产品的扩展可能会改变治疗的临床进程，并给许多患者带来新的机会。在这篇综述中，我们阐述了基因治疗的治疗可能性和递送方法，以及与之相关的挑战和不确定性。我们介绍了该领域正在研究的遗传性和获得性疾病，总结了眼部基因治疗的当前趋势，并指出了未来的发展方向。
