基因编辑技术是一项对基因组进行精确定点修饰的技术,可对特定DNA片段进行敲除、加入和替换等,从而在基因组水平上进行精确的基因编辑。其技术本质均是利用非同源末端链接途径修复和同源重组修复,联合特异性DNA靶向识别及核酸内切酶完成的DNA序列改变。基因编辑技术在科研、农业、医疗等领域都具有极其广泛的发展前景和应用价值。在疾病基因治疗领域,基因编辑技术在癌症如白血病、遗传性疾病如血友病、地中海贫血、多种肌肉营养障碍症和逆转录病毒相关传染病如艾滋病等疾病的治疗方面取得许多堪称跨时代的佳绩:结合基因编辑技术开展的实验新方法学研究及动物模型的制备工作也在迅猛地发展和完善;世界各地的实验室也已将基因编辑技术应用于预防疟疾、器官移植、生物制药、农业育种改良、灭绝物种复活等多个研究领域。该文就基因编辑技术在上述领域中的研究进展进行一些概括和总结。此外还归纳了一些当前针对基因编辑技术存在的争议和思考,并初步探讨了该技术在中药研究中的潜在应用前景。
Gene editing is a kind of technologies that makes precise modification to the genome. It can be used to knock out/in and replace the specific DNA fragment, and make accurate gene editing on the genome level. The essence of the technique is the DNA se- quence change with use of non homologous end link repair and homologous recombination repair, combined with specific DNA target recognition and endonuclease. This technology has wide range of development prospects and high application value in terms of scientific research, agriculture, medical treatment and other fields. In the field of gene therapy, gene editing technology has achieved cross-time success in cancers such as leukemia, genetic disorders such as hemophilia, thalassemia, multiple muscle nutritional disorders and ret- rovirus associated infectious diseases such as AIDS and other diseases. The preparation work for new experimental methods and animal models combined with gene editing technology is under rapid development and improvement. Laboratories around the world have also applied gene editing technique in prevention of malaria, organ transplantation, biological pharmaceuticals, agricultural breeding im- provement, resurrection of extinct species, and other research areas. This paper summarizes the application and development status of gene editing technique in the above fields, and also preliminarily explores the potential application prospect of the technology in the field of traditional Chinese medicine, and discusses the present controversy and thoughts.