Hemophilia Gene Therapy: New Development from Bench to Bed Side

被引：7
作者：
Guo, Xiao-Lu [1 ,4 ]
Chung, Tsai-Hua [1 ,2 ]
Qin, Yue [2 ]
Zheng, Jie [3 ]
Zheng, Huyong [3 ]
Sheng, Liyuan [4 ]
Wynn, Tung [5 ,6 ]
Chang, Lung-Ji [1 ,2 ,3 ,7 ]
机构：
[1] Genoimmune Med Inst, 2nd F1 Key Lab Bldg,Shenzhen Virtual Univ Pk 6, Shenzhen 518000, Guangdong, Peoples R China
[2] Univ Elect Sci & Technol China, Sch Med, Chengdu, Sichuan, Peoples R China
[3] Capital Med Univ, Beijing Childrens Hosp, Hematol Oncol Ctr, Natl Ctr Childrens Hlth, Beijing, Peoples R China
[4] PKU HKUST Shenzhen Hong Kong Inst, Shenzhen, Guangdong, Peoples R China
[5] Univ Florida, Dept Pediat, Gainesville, FL USA
[6] Univ Florida, Div Hematol Oncol, Gainesville, FL USA
[7] Univ Florida, Dept Mol Genet & Microbiol, Gainesville, FL USA
来源：
CURRENT GENE THERAPY | 2019年 / 19卷 / 04期
关键词：
Gene therapy; lentiviral vector; hemophilia; recessive disorder; novel gene therapy; hemostasis; FACTOR-VIII GENE; IMMUNE TOLERANCE INDUCTION; FACTOR-IX GENE; A MICE; CANINE HEMOPHILIA; EXPRESSION; DELIVERY; MODEL; CELLS; FVIII;
D O I：
10.2174/1566523219666190924121836
中图分类号：
Q3 [遗传学];
学科分类号：
071007 ; 090102 ;
摘要：
Novel gene therapy strategies have changed the prognosis of many inherited diseases in recent years. New development in genetic tools and study models has brought us closer to a complete cure for hemophilia. This review will address the latest gene therapy research in hemophilia A and B including gene therapy tools, genetic strategies and animal models. It also summarizes the results of recent clinical trials. Potential solutions are discussed regarding the current barriers in gene therapy for hemophilia.
引用
收藏
页码：264 / 273
页数：10
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