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Current animal models of hemophilia: the state of the art

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 t...

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Autores principales: Yen, Ching-Tzu, Fan, Meng-Ni, Yang, Yung-Li, Chou, Sheng-Chieh, Yu, I-Shing, Lin, Shu-Wha
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2016
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5056469/
https://www.ncbi.nlm.nih.gov/pubmed/27766048
http://dx.doi.org/10.1186/s12959-016-0106-0
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author Yen, Ching-Tzu
Fan, Meng-Ni
Yang, Yung-Li
Chou, Sheng-Chieh
Yu, I-Shing
Lin, Shu-Wha
author_facet Yen, Ching-Tzu
Fan, Meng-Ni
Yang, Yung-Li
Chou, Sheng-Chieh
Yu, I-Shing
Lin, Shu-Wha
author_sort Yen, Ching-Tzu
collection PubMed
description 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.
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spelling pubmed-50564692016-10-20 Current animal models of hemophilia: the state of the art Yen, Ching-Tzu Fan, Meng-Ni Yang, Yung-Li Chou, Sheng-Chieh Yu, I-Shing Lin, Shu-Wha Thromb J Review 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. BioMed Central 2016-10-04 /pmc/articles/PMC5056469/ /pubmed/27766048 http://dx.doi.org/10.1186/s12959-016-0106-0 Text en © The Author(s). 2016 Open AccessThis article is distributed under the terms of the Creative Commons Attribution 4.0 International License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated.
spellingShingle Review
Yen, Ching-Tzu
Fan, Meng-Ni
Yang, Yung-Li
Chou, Sheng-Chieh
Yu, I-Shing
Lin, Shu-Wha
Current animal models of hemophilia: the state of the art
title Current animal models of hemophilia: the state of the art
title_full Current animal models of hemophilia: the state of the art
title_fullStr Current animal models of hemophilia: the state of the art
title_full_unstemmed Current animal models of hemophilia: the state of the art
title_short Current animal models of hemophilia: the state of the art
title_sort current animal models of hemophilia: the state of the art
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5056469/
https://www.ncbi.nlm.nih.gov/pubmed/27766048
http://dx.doi.org/10.1186/s12959-016-0106-0
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