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Hemophilia Gene Therapy: From Bench to Bed Side.

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收录情况： ◇ SCIE

作者：

机构： [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]Hematology Oncology Center, Beijing Children's Hospital, Capital Medical University, National Center for Children`s Health, Beijing. China. [5]PKU-HKUST Shenzhen-Hong Kong Institution, Shenzhen. China. [6]Department of Pediatrics and Division of Hematology/Oncology, and fDepartment Molecular Genetics and Microbiology, University of Florida, Gainesville, Florida. United States.

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关键词： Gene therapy Hemophilia Hemostasis Lentiviral vector Novel gene therapy Recessive disorder

摘要：

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, and summarizes results of recent clinical trials. We will also discuss potential solutions to the current barriers in gene therapy for hemophilia. Copyright© Bentham Science Publishers; For any queries, please email at epub@benthamscience.net.

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中科院(CAS)分区：

出版当年[2018]版中科院分区表Review为是的期刊

：

大类 | 3 区医学

小类 | 4 区遗传学

最新[2023]版

：

大类 | 4 区医学

小类 | 4 区遗传学

JCR分区：

出版当年[2017]版：

Q3 GENETICS & HEREDITY

最新[2023]版：

Q2 GENETICS & HEREDITY

影响因子： 3.8 最新[2023版] 3.2 最新五年平均 1.943 出版当年[2017版] 2.225 出版当年五年平均 2.78 出版前一年[2016版] 2.218 出版后一年[2018版]

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第一作者机构： [1]Geno-immune Medical Institute, Shenzhen. China.

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