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General Review Article

Hemophilia Gene Therapy: New Development from Bench to Bed Side

Author(s): Xiao-Lu Guo, Tsai-Hua Chung, Yue Qin, Jie Zheng, Huyong Zheng, Liyuan Sheng, Tung Wynn and Lung-Ji Chang*

Volume 19, Issue 4, 2019

Page: [264 - 273] Pages: 10

DOI: 10.2174/1566523219666190924121836

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.

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