The use of genetically humanized animal models for personalized medicine approaches

For many genetic diseases, researchers are developing personalized medicine approaches. These sometimes employ custom genetic interventions such as antisense-mediated exon skipping or genome editing, aiming to restore protein function in a mutation-specific manner. Animal models can facilitate the d...

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Main Authors:	Annemieke Aartsma-Rus, Maaike van Putten, James Dowling
Format:	Article
Language:	English
Published:	The Company of Biologists 2020-02-01
Series:	Disease Models & Mechanisms
Subjects:	genetic therapies genome editing exon skipping pre-clinical studies muscular dystrophy
Online Access:	http://dmm.biologists.org/content/13/2/dmm041673

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spelling	doaj-9a55427efd994412af34cf2720b2636b2020-11-24T22:10:28ZengThe Company of BiologistsDisease Models & Mechanisms1754-84031754-84112020-02-0113210.1242/dmm.041673041673The use of genetically humanized animal models for personalized medicine approachesAnnemieke Aartsma-Rus0Maaike van Putten1Annemieke Aartsma-Rus2James Dowling3Maaike van Putten4 Department of Human Genetics, Leiden University Medical Center, Albinusdreef 2, 2333 ZA Leiden, the Netherlands Department of Human Genetics, Leiden University Medical Center, Albinusdreef 2, 2333 ZA Leiden, the Netherlands Department of Human Genetics, Leiden University Medical Center, Albinusdreef 2, 2333 ZA Leiden, the Netherlands Department of Human Genetics, Leiden University Medical Center, Albinusdreef 2, 2333 ZA Leiden, the Netherlands Department of Human Genetics, Leiden University Medical Center, Albinusdreef 2, 2333 ZA Leiden, the Netherlands For many genetic diseases, researchers are developing personalized medicine approaches. These sometimes employ custom genetic interventions such as antisense-mediated exon skipping or genome editing, aiming to restore protein function in a mutation-specific manner. Animal models can facilitate the development of personalized medicine approaches; however, given that they target human mutations and therefore human genetic sequences, scientists rely on the availability of humanized animal models. Here, we outline the usefulness, caveats and potential of such models, using the example of the hDMDdel52/mdx model, a humanized model recently generated for Duchenne muscular dystrophy (DMD).http://dmm.biologists.org/content/13/2/dmm041673genetic therapiesgenome editingexon skippingpre-clinical studiesmuscular dystrophy
collection	DOAJ
language	English
format	Article
sources	DOAJ
author	Annemieke Aartsma-Rus Maaike van Putten Annemieke Aartsma-Rus James Dowling Maaike van Putten
spellingShingle	Annemieke Aartsma-Rus Maaike van Putten Annemieke Aartsma-Rus James Dowling Maaike van Putten The use of genetically humanized animal models for personalized medicine approaches Disease Models & Mechanisms genetic therapies genome editing exon skipping pre-clinical studies muscular dystrophy
author_facet	Annemieke Aartsma-Rus Maaike van Putten Annemieke Aartsma-Rus James Dowling Maaike van Putten
author_sort	Annemieke Aartsma-Rus
title	The use of genetically humanized animal models for personalized medicine approaches
title_short	The use of genetically humanized animal models for personalized medicine approaches
title_full	The use of genetically humanized animal models for personalized medicine approaches
title_fullStr	The use of genetically humanized animal models for personalized medicine approaches
title_full_unstemmed	The use of genetically humanized animal models for personalized medicine approaches
title_sort	use of genetically humanized animal models for personalized medicine approaches
publisher	The Company of Biologists
series	Disease Models & Mechanisms
issn	1754-8403 1754-8411
publishDate	2020-02-01
description	For many genetic diseases, researchers are developing personalized medicine approaches. These sometimes employ custom genetic interventions such as antisense-mediated exon skipping or genome editing, aiming to restore protein function in a mutation-specific manner. Animal models can facilitate the development of personalized medicine approaches; however, given that they target human mutations and therefore human genetic sequences, scientists rely on the availability of humanized animal models. Here, we outline the usefulness, caveats and potential of such models, using the example of the hDMDdel52/mdx model, a humanized model recently generated for Duchenne muscular dystrophy (DMD).
topic	genetic therapies genome editing exon skipping pre-clinical studies muscular dystrophy
url	http://dmm.biologists.org/content/13/2/dmm041673
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The use of genetically humanized animal models for personalized medicine approaches

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