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

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

Molecular Genetics and Microbiology

Research output: Contribution to journal › Article › peer-review

8 Citations (Scopus)

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.

Original language	English
Pages (from-to)	264-273
Number of pages	10
Journal	Current Gene Therapy
Volume	19
Issue number	4
DOIs	https://doi.org/10.2174/1566523219666190924121836
Publication status	Published - 2019

Bibliographical note

Publisher Copyright:
© 2019 Bentham Science Publishers.

ASJC Scopus Subject Areas

Molecular Medicine
Molecular Biology
Genetics
Drug Discovery
Genetics(clinical)

Keywords

Gene therapy
Hemophilia
Hemostasis
Lentiviral vector
Novel gene therapy
Recessive disorder

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10.2174/1566523219666190924121836

Cite this

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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.",

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AU - Guo, Xiao Lu

AU - Chung, Tsai Hua

AU - Qin, Yue

AU - Zheng, Jie

AU - Zheng, Huyong

AU - Sheng, Liyuan

AU - Wynn, Tung

AU - Chang, Lung Ji

PY - 2019

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AB - 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.

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KW - Hemophilia

KW - Hemostasis

KW - Lentiviral vector

KW - Novel gene therapy

KW - Recessive disorder

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Hemophilia gene therapy: New development from bench to bed side

Abstract

Bibliographical note

ASJC Scopus Subject Areas

Keywords

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