Characterization of dystrophin deficient rats: a new model for Duchenne muscular dystrophy.

A few animal models of Duchenne muscular dystrophy (DMD) are available, large ones such as pigs or dogs being expensive and difficult to handle. Mdx (X-linked muscular dystrophy) mice only partially mimic the human disease, with limited chronic muscular lesions and muscle weakness. Their small size...

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Autores principales:	Thibaut Larcher, Aude Lafoux, Laurent Tesson, Séverine Remy, Virginie Thepenier, Virginie François, Caroline Le Guiner, Helicia Goubin, Maéva Dutilleul, Lydie Guigand, Gilles Toumaniantz, Anne De Cian, Charlotte Boix, Jean-Baptiste Renaud, Yan Cherel, Carine Giovannangeli, Jean-Paul Concordet, Ignacio Anegon, Corinne Huchet
Formato:	article
Lenguaje:	EN
Publicado:	Public Library of Science (PLoS) 2014
Materias:	Medicine R Science Q
Acceso en línea:	https://doaj.org/article/5360c456b3714a1faaa7b80cda6608fc
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spelling	oai:doaj.org-article:5360c456b3714a1faaa7b80cda6608fc2021-11-25T05:56:50ZCharacterization of dystrophin deficient rats: a new model for Duchenne muscular dystrophy.1932-620310.1371/journal.pone.0110371https://doaj.org/article/5360c456b3714a1faaa7b80cda6608fc2014-01-01T00:00:00Zhttps://doi.org/10.1371/journal.pone.0110371https://doaj.org/toc/1932-6203A few animal models of Duchenne muscular dystrophy (DMD) are available, large ones such as pigs or dogs being expensive and difficult to handle. Mdx (X-linked muscular dystrophy) mice only partially mimic the human disease, with limited chronic muscular lesions and muscle weakness. Their small size also imposes limitations on analyses. A rat model could represent a useful alternative since rats are small animals but 10 times bigger than mice and could better reflect the lesions and functional abnormalities observed in DMD patients. Two lines of Dmd mutated-rats (Dmdmdx) were generated using TALENs targeting exon 23. Muscles of animals of both lines showed undetectable levels of dystrophin by western blot and less than 5% of dystrophin positive fibers by immunohistochemistry. At 3 months, limb and diaphragm muscles from Dmdmdx rats displayed severe necrosis and regeneration. At 7 months, these muscles also showed severe fibrosis and some adipose tissue infiltration. Dmdmdx rats showed significant reduction in muscle strength and a decrease in spontaneous motor activity. Furthermore, heart morphology was indicative of dilated cardiomyopathy associated histologically with necrotic and fibrotic changes. Echocardiography showed significant concentric remodeling and alteration of diastolic function. In conclusion, Dmdmdx rats represent a new faithful small animal model of DMD.Thibaut LarcherAude LafouxLaurent TessonSéverine RemyVirginie ThepenierVirginie FrançoisCaroline Le GuinerHelicia GoubinMaéva DutilleulLydie GuigandGilles ToumaniantzAnne De CianCharlotte BoixJean-Baptiste RenaudYan CherelCarine GiovannangeliJean-Paul ConcordetIgnacio AnegonCorinne HuchetPublic Library of Science (PLoS)articleMedicineRScienceQENPLoS ONE, Vol 9, Iss 10, p e110371 (2014)
institution	DOAJ
collection	DOAJ
language	EN
topic	Medicine R Science Q
spellingShingle	Medicine R Science Q Thibaut Larcher Aude Lafoux Laurent Tesson Séverine Remy Virginie Thepenier Virginie François Caroline Le Guiner Helicia Goubin Maéva Dutilleul Lydie Guigand Gilles Toumaniantz Anne De Cian Charlotte Boix Jean-Baptiste Renaud Yan Cherel Carine Giovannangeli Jean-Paul Concordet Ignacio Anegon Corinne Huchet Characterization of dystrophin deficient rats: a new model for Duchenne muscular dystrophy.
description	A few animal models of Duchenne muscular dystrophy (DMD) are available, large ones such as pigs or dogs being expensive and difficult to handle. Mdx (X-linked muscular dystrophy) mice only partially mimic the human disease, with limited chronic muscular lesions and muscle weakness. Their small size also imposes limitations on analyses. A rat model could represent a useful alternative since rats are small animals but 10 times bigger than mice and could better reflect the lesions and functional abnormalities observed in DMD patients. Two lines of Dmd mutated-rats (Dmdmdx) were generated using TALENs targeting exon 23. Muscles of animals of both lines showed undetectable levels of dystrophin by western blot and less than 5% of dystrophin positive fibers by immunohistochemistry. At 3 months, limb and diaphragm muscles from Dmdmdx rats displayed severe necrosis and regeneration. At 7 months, these muscles also showed severe fibrosis and some adipose tissue infiltration. Dmdmdx rats showed significant reduction in muscle strength and a decrease in spontaneous motor activity. Furthermore, heart morphology was indicative of dilated cardiomyopathy associated histologically with necrotic and fibrotic changes. Echocardiography showed significant concentric remodeling and alteration of diastolic function. In conclusion, Dmdmdx rats represent a new faithful small animal model of DMD.
format	article
author	Thibaut Larcher Aude Lafoux Laurent Tesson Séverine Remy Virginie Thepenier Virginie François Caroline Le Guiner Helicia Goubin Maéva Dutilleul Lydie Guigand Gilles Toumaniantz Anne De Cian Charlotte Boix Jean-Baptiste Renaud Yan Cherel Carine Giovannangeli Jean-Paul Concordet Ignacio Anegon Corinne Huchet
author_facet	Thibaut Larcher Aude Lafoux Laurent Tesson Séverine Remy Virginie Thepenier Virginie François Caroline Le Guiner Helicia Goubin Maéva Dutilleul Lydie Guigand Gilles Toumaniantz Anne De Cian Charlotte Boix Jean-Baptiste Renaud Yan Cherel Carine Giovannangeli Jean-Paul Concordet Ignacio Anegon Corinne Huchet
author_sort	Thibaut Larcher
title	Characterization of dystrophin deficient rats: a new model for Duchenne muscular dystrophy.
title_short	Characterization of dystrophin deficient rats: a new model for Duchenne muscular dystrophy.
title_full	Characterization of dystrophin deficient rats: a new model for Duchenne muscular dystrophy.
title_fullStr	Characterization of dystrophin deficient rats: a new model for Duchenne muscular dystrophy.
title_full_unstemmed	Characterization of dystrophin deficient rats: a new model for Duchenne muscular dystrophy.
title_sort	characterization of dystrophin deficient rats: a new model for duchenne muscular dystrophy.
publisher	Public Library of Science (PLoS)
publishDate	2014
url	https://doaj.org/article/5360c456b3714a1faaa7b80cda6608fc
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Characterization of dystrophin deficient rats: a new model for Duchenne muscular dystrophy.

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