基因治疗发展现状及展望
投稿时间:2021-12-06  修订日期:2022-07-06  点此下载全文
引用本文:史彤,李积宗.基因治疗发展现状及展望[J].药学实践杂志,2022,40(4):296~301,313
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作者单位E-mail
史彤 上海市生物医药科技发展中心上海 201203  
李积宗 上海市生物医药科技发展中心上海 201203 jizongli@stcsm.sh.gov.cn 
基金项目:上海市生物医药产业技术创新服务平台”专项(20DZ2292100)
中文摘要:基因治疗是指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目的的治疗手段。随着载体递送、基因编辑等相关技术的快速发展,基因治疗已被广泛应用于遗传性疾病和肿瘤等难治性疾病的研究,其基因产品在多个国家获批上市,显示出令人惊喜的疗效。大型药企纷纷布局基因治疗领域,同时涌现出大量创新型企业,加入到基因治疗研发的行列,基因治疗展现出广阔的市场前景。本文从基因治疗关键技术的研究进展、产业发展、监管政策及未来展望等方面进行探讨。
中文关键词:基因治疗  基因编辑  产业发展  监管政策
 
Progress and perspective of gene therapy
Abstract:Gene therapy is generally referred as a therapeutic method through modifying or manipulating gene expression and accordingly changing biological characteristics of living cells. With the substantial progress of vector delivery, gene editing and other relevant technologies, gene therapy has been widely applied in the research of genetic disorder, cancer and other refractory diseases. Gene therapy products have been approved in many countries and exhibited amazing therapeutic effectiveness. Big pharmaceutical companies have strategically entered this field along with many innovative entrepreneurs. Gene therapy has showed great market potential and prospect. In this review, research progresses, industrial developments, administration policies, as well as future expectations of the gene therapy were discussed.
keywords:gene therapy  gene editing  industrial development  administration policy
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