视网膜相关疾病基因治疗的现状

The status quo of gene therapy for retinal related disease

随着对各种疾病认识的深入和医疗手段的提高,精准医学要求我们将研究细化到分子水平。基因治疗以其独有的优势成为遗传性疾病的重要治疗手段。视网膜由于其特有的优势一直处在基因治疗研究的最前沿,已在体外实验和疾病模型中获得了较为成熟的理论和技术,已有部分转化为临床成果。本文重点综述视网膜相关疾病的基因治疗现状,分析其研究成果、突变位点和疾病模型,希望对后续疾病的研究方向和治疗手段提供有价值的参考。我们还列出了目前常用的基因治疗载体,以供相关研究者选择和改进。

With the thorough knowledge of various diseases and advanced approach of medical treatment,precision medicine demands to refine to molecular level.Gene therapy,with its advantages,has been increasingly becoming a critical method of inherited and acquired diseases.Due to its unique advantages,retina has been at the forefront of gene therapy research,and has obtained relatively mature theories and technologies in in vitro experiments and disease models,some of them have already transformed into clinical results.Here,we discuss the status quo of several retinal researches,analyzing their research results,mutation sites and disease models,hoping to provide a valuable reference for the direction and treatment of subsequent researches.We also persent a list of commonly used gene therapy vectors for the selection and improvement of relevant researchers.