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Muscular dystrophy: Experimental animal models and therapeutic approaches (Review)

The muscular dystrophies are a heterogeneous group of genetically inherited diseases characterized by muscle weakness and progressive wasting, which can cause premature death in severe forms. Although >30 years have passed since the identification of the first protein involved in a type of muscul...

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Autores principales: Gaina, Gisela, Popa (Gruianu), Alexandra
Formato: Online Artículo Texto
Lenguaje:English
Publicado: D.A. Spandidos 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8082581/
https://www.ncbi.nlm.nih.gov/pubmed/33936267
http://dx.doi.org/10.3892/etm.2021.10042
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author Gaina, Gisela
Popa (Gruianu), Alexandra
author_facet Gaina, Gisela
Popa (Gruianu), Alexandra
author_sort Gaina, Gisela
collection PubMed
description The muscular dystrophies are a heterogeneous group of genetically inherited diseases characterized by muscle weakness and progressive wasting, which can cause premature death in severe forms. Although >30 years have passed since the identification of the first protein involved in a type of muscular dystrophy, there is no effective treatment for these disabling disorders. In the last decade, several novel therapeutic approaches have been developed and investigated as promising therapeutic approaches aimed to ameliorate the dystrophic phenotype either by restoring dystrophin expression or by compensating for dystrophin deficiency. Concurrently, with the development of therapeutic approaches, in addition to naturally occurring animal models, a wide range of genetically engineered animal models has been generated. The use of animals as models of muscular dystrophies has greatly improved the understanding of the pathogenicity of these diseases and has proven useful in gene therapy studies. In this review, we summarize these latest innovative therapeutic approaches to muscular dystrophies and the usefulness of the various most common experimental animal models.
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spelling pubmed-80825812021-04-30 Muscular dystrophy: Experimental animal models and therapeutic approaches (Review) Gaina, Gisela Popa (Gruianu), Alexandra Exp Ther Med Review The muscular dystrophies are a heterogeneous group of genetically inherited diseases characterized by muscle weakness and progressive wasting, which can cause premature death in severe forms. Although >30 years have passed since the identification of the first protein involved in a type of muscular dystrophy, there is no effective treatment for these disabling disorders. In the last decade, several novel therapeutic approaches have been developed and investigated as promising therapeutic approaches aimed to ameliorate the dystrophic phenotype either by restoring dystrophin expression or by compensating for dystrophin deficiency. Concurrently, with the development of therapeutic approaches, in addition to naturally occurring animal models, a wide range of genetically engineered animal models has been generated. The use of animals as models of muscular dystrophies has greatly improved the understanding of the pathogenicity of these diseases and has proven useful in gene therapy studies. In this review, we summarize these latest innovative therapeutic approaches to muscular dystrophies and the usefulness of the various most common experimental animal models. D.A. Spandidos 2021-06 2021-04-14 /pmc/articles/PMC8082581/ /pubmed/33936267 http://dx.doi.org/10.3892/etm.2021.10042 Text en Copyright: © Gaina et al. https://creativecommons.org/licenses/by-nc-nd/4.0/This is an open access article distributed under the terms of the Creative Commons Attribution-NonCommercial-NoDerivs License (https://creativecommons.org/licenses/by-nc-nd/4.0/) , which permits use and distribution in any medium, provided the original work is properly cited, the use is non-commercial and no modifications or adaptations are made.
spellingShingle Review
Gaina, Gisela
Popa (Gruianu), Alexandra
Muscular dystrophy: Experimental animal models and therapeutic approaches (Review)
title Muscular dystrophy: Experimental animal models and therapeutic approaches (Review)
title_full Muscular dystrophy: Experimental animal models and therapeutic approaches (Review)
title_fullStr Muscular dystrophy: Experimental animal models and therapeutic approaches (Review)
title_full_unstemmed Muscular dystrophy: Experimental animal models and therapeutic approaches (Review)
title_short Muscular dystrophy: Experimental animal models and therapeutic approaches (Review)
title_sort muscular dystrophy: experimental animal models and therapeutic approaches (review)
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8082581/
https://www.ncbi.nlm.nih.gov/pubmed/33936267
http://dx.doi.org/10.3892/etm.2021.10042
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