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科技导报
| 综述 2020, 38(15): 89-100
单基因遗传疾病的基因疗法
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周丽娟1,2 , 王京春1 , 高宏斌1 , 赵东平1,2
作者信息
1. 中国科普研究所, 北京 100081;
通讯作者:
王京春(通信作者),研究员,研究方向为科学传播及科学素质测评,电子信箱:wangjingchun@cstm.org.cn
Gene therapy for monogenetic diseases
Affiliations
出版时间: 2020-08-13
doi: 10.3981/j.issn.1000-7857.2020.15.010
文章导航
基因疗法是全球突破性技术之一，在单基因遗传疾病治疗中已取得突破性进展。阐述了不同的基因疗法策略、载体和基因编辑技术的特点，综述了脊髓性肌萎缩症、Leber先天性黑蒙2型、血友病、β -地中海贫血的发病机理、临床表现、基因疗法的开发进程以及临床试验情况。目前，上述4种单基因疾病的9种基因疗法已分别取得美国食品药品监督管理局突破性疗法资格、欧洲药品管理局的优先药物资格或者已经批准上市。基因疗法的研究还面临着许多挑战，但随着科学研究的深入和科学技术的不断发展，将有更多的患者获得治疗。
基因疗法
/
脊髓型肌萎缩
/
Leber先天性黑蒙2型
/
血友病
/
β-地中海贫血
Gene therapy is one of the world's breakthrough technologies, which has made breakthrough progress in the treatment of monogenic diseases. In this article the characteristics of different gene therapy strategies, vectors and gene editing techniques are described, with focuses on reviewing pathogenesis, clinical manifestations, development process of gene therapy and clinical trials of spinal muscular atrophy (SMA), Leber congenital amaurosis type 2 (LCA2), hemophilia, and β -thalassemia. At present, 9 gene therapies of the above 4 monogenic diseases have earned "breakthrough therapy" designation by the U.S. Food and Drug Administration (FDA), PRIME designation by the European Medicines Agency (EMA) or have been the gene therapies available on the market. The research of gene therapy faces many challenges but with the development of science and technology more patients will get treatment.
gene therapy
/
spinal muscular atrophy
/
Leber congenital amaurosis type 2
/
hemophilia
/
β-thalassemia
周丽娟, 王京春, 高宏斌, 赵东平.
单基因遗传疾病的基因疗法.
科技导报,
2020
, 38
(15)
: 89
-100
.
DOI: 10.3981/j.issn.1000-7857.2020.15.010
ZHOU Lijuan, WANG Jingchun, GAO Hongbin, ZHAO Dongping.
Gene therapy for monogenetic diseases[J].
Science & Technology Review ,
2020
, 38
(15)
: 89
-100
.
DOI: 10.3981/j.issn.1000-7857.2020.15.010
2020年第38卷第15期
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文章信息
doi: 10.3981/j.issn.1000-7857.2020.15.010
接收时间：2020-03-18
首发时间：2020-08-14
出版时间：2020-08-13
收稿日期：2020-03-18
修回日期：2020-05-21
通讯作者:
王京春(通信作者),研究员,研究方向为科学传播及科学素质测评,电子信箱:wangjingchun@cstm.org.cn
https://castjournals.cast.org.cn/joweb/kjdb/CN/10.3981/j.issn.1000-7857.2020.15.010
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2种不同金属材料的力学参数
科 属数 种数 占总种数比例 属 种数 占总种数比例 鹅膏菌科Amanitaceae 2 11 5.26 鹅膏菌属 Amanita 10 4.78 小菇科 Mycenaceae 2 12 5.74 丝盖伞属 Inocybe 5 2.39 多孔菌科 Polyporaceae 8 14 6.70 蜡蘑属 Laccaria 5 2.39 红菇科 Russulaceae 3 23 11.00 小皮伞属 Marasmius 6 2.87 小菇属 Mycena 11 5.26 光柄菇属 Pluteus 5 2.39 红菇属 Russula 17 8.13 栓菌属 Trametes 5 2.39
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