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The approved gene therapy drugs worldwide: from 1998 to 2019.
Biotechnology Advances ( IF 12.1 ) Pub Date : 2019-12-27 , DOI: 10.1016/j.biotechadv.2019.107502
Cui-Cui Ma 1 , Zhen-Ling Wang 1 , Ting Xu 2 , Zhi-Yao He 2 , Yu-Quan Wei 1
Affiliation  

With the improvement of gene vectors, the rise of chimeric antigen receptor T cell immunotherapy and breakthroughs in the genome editing technology, gene therapy had once again returned to the central stage of disease treatment. It had brought new choices to clinical therapy of diseases such as tumors and genetic diseases, and had changed the status quo of treatment for monogenic disorders and diffuse large B-cell lymphoma. Until August 2019, 22 gene medicines had been approved by the drug regulatory agencies from various countries, but there were few relevant reviews of combing these drugs systematically. Consequently, this review summarizes the gene therapy drugs approved worldwide from 1998 to 2019 in details, including names, indications, dates of approval, companies, vectors, the applied technologies and mechanisms of gene therapy drugs, etc. Furthermore, the gene therapy drugs were classified and addressed in accordance with the employed vectors. Gene therapy had gradually been accepted by the government and the public since 1980s, and have become a new and important alternative to existing treatments for human diseases in the past few years. Therefore, gene therapy drugs, with safe vectors and advanced biotechnologies, would play a greater role in the prevention and treatment of human diseases in future.



中文翻译:

1998年至2019年,全球批准的基因治疗药物。

随着基因载体的改进,嵌合抗原受体T细胞免疫治疗的兴起以及基因组编辑技术的突破,基因治疗再次回到疾病治疗的中心阶段。它为肿瘤和遗传疾病等疾病的临床治疗带来了新的选择,并改变了单基因疾病和弥漫性大B细胞淋巴瘤的治疗现状。直到2019年8月,各国药物监管机构已经批准了22种基因药物,但是对于系统地组合这些药物的相关评论很少。因此,本综述详细总结了1998年至2019年全球批准的基因治疗药物,包括名称,适应症,批准日期,公司,载体,基因治疗药物的应用技术和机理等。此外,根据所使用的载体对基因治疗药物进行分类和处理。自1980年代以来,基因疗法已逐渐为政府和公众所接受,并且在过去几年中已成为现有的人类疾病疗法的新的重要替代方法。因此,具有安全载体和先进生物技术的基因治疗药物将在未来人类疾病的预防和治疗中发挥更大的作用。

更新日期:2019-12-27
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