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1990年基因治疗最早应用于临床。随着人类基因组研究计划的成功,基因图及基因序列的完成,将使我们得以在基因水平认识疾病的发生,发展。应用针对某一变异基因的特异性药物以及替代病变的基因进行基因治疗将成为极具潜力的临床治疗方法;同时,通过基因治疗还可将新的功能导入细胞。外源基因的导入可以通过病毒载体或非病毒载体来实现。就基因治疗的内容、方法及在眼科方面的研究及应用前景进行综述。
Gene therapy was first applied to clinical in 1990. With the success of the human genome research project, the completion of the genetic map and gene sequence will enable us to recognize the occurrence and development of the disease at the gene level. Gene therapy with specific agents for a particular mutation and genes that substitute for disease will be a potential clinical cure; at the same time, new functions can be introduced into cells through gene therapy. Introduction of exogenous genes can be achieved by viral vectors or non-viral vectors. This review summarizes the contents and methods of gene therapy and the research and application prospect in ophthalmology.