基因治疗产品及其载体的研究进展和挑战

Research Progress and Challenges of Gene Therapy Products and Vectors

基因治疗是一种通过改变个体的基因表达来治疗疾病的方法,为肿瘤、罕见病及其他难治性疾病提供了全新的治疗策略。基因治疗通过基因编辑技术实现对个体致病基因的改造,同时也依赖基因递送载体改善其在体内的稳定性和靶向性。本综述简述了基因治疗的方式,结合目前的基因治疗产品,介绍了病毒、非病毒基因递送载体和基因编辑技术在基因治疗中的发展概况,并总结归纳了临床实践中基因治疗的安全性问题。

Gene therapy is a new approach to treating diseases by altering gene expression in vivo,provides a new treatment strategy for tumors,rare diseases,and other refractory diseases.Gene therapy modifies the pathogenic genes of an individual through gene editing technology and relies on gene delivery vectors to improve the stability and targeting in vivo of genes.This review briefly describes gene therapy methods,introduces the development of viral and non-viral vectors for gene delivery and gene editing technologies in gene therapy combined with the current gene therapy products,and summarizes the safety issues of gene therapy in clinical practice.