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

Producción científica: Contribución a una revista › Artículo › revisión exhaustiva

8 Citas (Scopus)

Resumen

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.

Idioma original	Inglés
Páginas (desde-hasta)	264-273
Número de páginas	10
Publicación	Current Gene Therapy
Volumen	19
N.º	4
DOI	https://doi.org/10.2174/1566523219666190924121836
Estado	Publicada - 2019

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Publisher Copyright:
© 2019 Bentham Science Publishers.

!!!ASJC Scopus Subject Areas

Medicina molecular
Biología molecular
Genética
Descubrimiento de medicamentos
Genética (clínica)

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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

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AU - Zheng, Huyong

AU - Sheng, Liyuan

AU - Wynn, Tung

AU - Chang, Lung Ji

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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 - Recessive disorder

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Hemophilia gene therapy: New development from bench to bed side

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