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Hemophilia Gene Therapy: New Development from Bench to Bed Side

[ Vol. 19 , Issue. 4 ]

Author(s):

Xiao-Lu Guo, Tsai-Hua Chung, Yue Qin, Jie Zheng, Huyong Zheng, Liyuan Sheng, Tung Wynn and Lung-Ji Chang*   Pages 264 - 273 ( 10 )

Abstract:


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.

Keywords:

Gene therapy, lentiviral vector, hemophilia, recessive disorder, novel gene therapy, hemostasis.

Affiliation:

Geno-immune Medical Institute, Shenzhen, Geno-immune Medical Institute, Shenzhen, School of Medicine, University of Electronic Science and Technology of China, Sichuan, Hematology Oncology Center, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, Hematology Oncology Center, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, PKU-HKUST Shenzhen-Hong Kong Institution, Shenzhen, Department of Pediatrics and Division of Hematology/Oncology, University of Florida, Gainesville, FL, Geno-immune Medical Institute, Shenzhen

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