Hemophilia Gene Therapy: New Development from Bench to Bed Side

Xiao-Lu Guo; Tsai-Hua Chung; Yue Qin; Jie Zheng; Huyong Zheng; Liyuan Sheng; Tung Wynn; Lung-Ji Chang

doi:10.2174/1566523219666190924121836

Hemophilia Gene Therapy: New Development from Bench to Bed Side

Curr Gene Ther. 2019;19(4):264-273. doi: 10.2174/1566523219666190924121836.

Authors

Xiao-Lu Guo^{1

2}, Tsai-Hua Chung^{1

3}, Yue Qin³, Jie Zheng⁴, Huyong Zheng⁴, Liyuan Sheng², Tung Wynn⁵, Lung-Ji Chang^{1

3

4

6}

Affiliations

¹ Geno-immune Medical Institute, Shenzhen, China.
² PKU-HKUST Shenzhen-Hong Kong Institution, Shenzhen, China.
³ School of Medicine, University of Electronic Science and Technology of China, Sichuan, China.
⁴ Hematology Oncology Center, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China.
⁵ Department of Pediatrics and Division of Hematology/Oncology, University of Florida, Gainesville, FL, United States.
⁶ Department of Molecular Genetics and Microbiology, University of Florida, Gainesville, FL, United States.

PMID: 31549954
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; hemophilia; hemostasis; lentiviral vector; novel gene therapy; recessive disorder..

Publication types

Research Support, Non-U.S. Gov't
Review

MeSH terms

Animals
Dependovirus / genetics*
Genetic Therapy*
Genetic Vectors / administration & dosage*
Hemophilia A / genetics*
Hemophilia A / therapy*
Humans
Translational Research, Biomedical*