《生命科学》 2023, 35(1): 95-102

2022年基因治疗领域发展态势

杨若南 , 许　丽 , 李　伟 , 徐　萍^*

中国科学院上海营养与健康研究所，中国科学院上海生命科学信息中心，上海 200031

摘　要：

基因治疗是通过修饰或操纵基因的表达，从而改变活细胞的生物学特性，以达到治疗疾病的目的。基因治疗已成为生物医药领域继小分子、大分子之后的一条热门新赛道。本文对2022 年全球基因治疗领域的政策规划、研究进展进行了总结。分析发现，载体递送及基因编辑等技术的突破加速基因治疗发展，基因治疗应用愈加广泛，适应证已从遗传性疾病扩展到癌症、心血管疾病等领域，获批产品不断增多，基因治疗产业化加速发展。此外，本文也对基因治疗发展趋势进行了展望。

通讯作者：徐　萍 , Email:xuping@sinh.ac.cn

The development of gene therapy in 2022

YANG Ruo-Nan , XU Li , LI Wei , XU Ping^*

(Shanghai Information Center for Life Sciences, Shanghai Institute of Nutrition and Health, Chinese Academy of Sciences, Shanghai 200031, China

Abstract:

Gene therapy refers to the treatment of diseases by modifying or manipulating the expression of genes, or altering the biological characteristics of living cells. Gene therapy has become a new important research direction in the field of biomedicine and biopharmaceutical. This review summarizes the policy planning and research progress in the field of gene therapy worldwide in 2022. It shows that the breakthrough of drug delivery system and gene editing technology accelerates the development of gene therapy, and the application of gene therapy is more and more extensive. The indications have been extended from genetic diseases to cancer and cardiovascular disease and other conditions. The number of approved products is increasing, and the industrialization of gene therapy is accelerating. The development trend of gene therapy is also prospected in this review.

Communication Author：XU Ping , Email:xuping@sinh.ac.cn