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Current animal models of hemophilia: the state of the art.

作者信息

Yen Ching-Tzu, Fan Meng-Ni, Yang Yung-Li, Chou Sheng-Chieh, Yu I-Shing, Lin Shu-Wha

机构信息

Department of Clinical Laboratory Science and Medical Biotechnology, College of Medicine, National Taiwan University, Taipei, Taiwan ; Center of Genomic Medicine, National Taiwan University, Taipei, Taiwan.

Department of Clinical Laboratory Science and Medical Biotechnology, College of Medicine, National Taiwan University, Taipei, Taiwan.

出版信息

Thromb J. 2016 Oct 4;14(Suppl 1):22. doi: 10.1186/s12959-016-0106-0. eCollection 2016.


DOI:10.1186/s12959-016-0106-0
PMID:27766048
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC5056469/
Abstract

Hemophilia is the most well-known hereditary bleeding disorder, with an incidence of one in every 5000 to 30,000 males worldwide. The disease is treated by infusion of protein products on demand and as prophylaxis. Although these therapies have been very successful, some challenging and unresolved tasks remain, such as reducing bleeding rates, presence of target joints and/or established joint damage, eliminating the development of inhibitors, and increasing the success rate of immune-tolerance induction (ITI). Many preclinical trials are carried out on animal models for hemophilia generated by the hemophilia research community, which in turn enable prospective clinical trials aiming to tackle these challenges. Suitable animal models are needed for greater advances in treating hemophilia, such as the development of better models for evaluation of the efficacy and safety of long-acting products, more powerful gene therapy vectors than are currently available, and successful ITI strategies. Mice, dogs, and pigs are the most commonly used animal models for hemophilia. With the advent of the nuclease method for genome editing, namely the CRISPR/Cas9 system, it is now possible to create animal models for hemophilia other than mice in a short period of time. This review presents currently available animal models for hemophilia, and discusses the importance of animal models for the development of better treatment options for hemophilia.

摘要
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/700d/5056469/d3aec9b494eb/12959_2016_106_Fig1_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/700d/5056469/d3aec9b494eb/12959_2016_106_Fig1_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/700d/5056469/d3aec9b494eb/12959_2016_106_Fig1_HTML.jpg

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[5]
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[6]
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[7]
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[9]
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本文引用的文献

[1]
Prophylactic efficacy of BeneFIX vs Alprolix in hemophilia B mice.

Blood. 2016-7-14

[2]
Genetic Targeting of the Albumin Locus to Treat Hemophilia.

N Engl J Med. 2016-3-31

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Canine models of inherited bleeding disorders in the development of coagulation assays, novel protein replacement and gene therapies.

J Thromb Haemost. 2016-4-5

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J Thromb Haemost. 2016-2

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J Thromb Haemost. 2015-6

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Optimized human factor IX expression cassettes for hepatic-directed gene therapy of hemophilia B.

Front Med. 2015-3

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Blood. 2015-3-5

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Hum Gene Ther. 2015-2

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Recombinant factor VIII products and inhibitor development in previously untreated boys with severe hemophilia A.

Blood. 2014-9-24

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Development and applications of CRISPR-Cas9 for genome engineering.

Cell. 2014-6-5

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