Hemophilia Gene Therapy: New Development from Bench to Bed Side-Reference-Cited by-同舟云学术

Hemophilia Gene Therapy: New Development from Bench to Bed Side

Published:2019-11-18 Issue:4 Volume:19 Page:264-273
ISSN:1566-5232
Container-title:Current Gene Therapy
language:en
Short-container-title:CGT

Author:

Guo Xiao-Lu¹,Chung Tsai-Hua¹,Qin Yue²,Zheng Jie³,Zheng Huyong³^ORCID,Sheng Liyuan⁴,Wynn Tung⁵,Chang Lung-Ji¹

Affiliation:

1. Geno-immune Medical Institute, Shenzhen, China

2. School of Medicine, University of Electronic Science and Technology of China, Sichuan, China

3. Hematology Oncology Center, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China

4. PKU-HKUST Shenzhen-Hong Kong Institution, Shenzhen, China

5. Department of Pediatrics and Division of Hematology/Oncology, University of Florida, Gainesville, FL, United States

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.

Funder

Natural Science Foundation of Guangdong Province

University of Electronic Science and Technology of China

Science and Technology Planning Technical Research Project of Shenzhen

Publisher

Bentham Science Publishers Ltd.

Subject

Genetics (clinical),Drug Discovery,Genetics,Molecular Biology,Molecular Medicine

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