基因治疗在临床应用中的研究进展

Research progress of gene therapy in clinical application

摘要:

20世纪60年代，科学家首次提出利用基因治疗治愈遗传疾病的概念。这一全新的概念性策略旨在通过将外源性遗传物质引入患者体内来获得长期的治疗效果。五十年的风雨沉浮，21世纪取得的里程碑式突破为基因治疗开启了新的篇章。文中回顾和总结了基因治疗的发展历程和重大突破，包括一些重要的临床试验和已批准上市的基因疗法，以及新兴的基因编辑技术。相对于传统疗法的独特优势，基因疗法将会成为治疗遗传疾病的重要手段，必将造福全人类。

Abstract:

In the 1960s, scientists first raised the idea of curing genetic diseases using gene therapy. This new conceptual strategy aimed to achieve a much longer therapeutic effect by introducing exogenous genetic materials into the patients. After more than five decades of ups and downs, gene therapy has been brought into a new era by those milestone breakthroughs in the 21st century. Here we reviewed and summarized the history and breakthroughs of gene therapy, including some critical clinical trials, approved drugs, and emerging gene editing techniques. We believe that with their unique advantages over traditional therapies, more gene therapies will become practical approaches to genetic diseases and benefit the entire human race.