Open Access

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

  • Authors:
    • Gisela Gaina
    • Alexandra Popa (Gruianu)
  • View Affiliations

  • Published online on: April 14, 2021     https://doi.org/10.3892/etm.2021.10042
  • Article Number: 610
  • Copyright: © Gaina et al. This is an open access article distributed under the terms of Creative Commons Attribution License.

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Abstract

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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June-2021
Volume 21 Issue 6

Print ISSN: 1792-0981
Online ISSN:1792-1015

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Spandidos Publications style
Gaina G and Gaina G: Muscular dystrophy: Experimental animal models and therapeutic approaches (Review). Exp Ther Med 21: 610, 2021
APA
Gaina, G., & Gaina, G. (2021). Muscular dystrophy: Experimental animal models and therapeutic approaches (Review). Experimental and Therapeutic Medicine, 21, 610. https://doi.org/10.3892/etm.2021.10042
MLA
Gaina, G., Popa (Gruianu), A."Muscular dystrophy: Experimental animal models and therapeutic approaches (Review)". Experimental and Therapeutic Medicine 21.6 (2021): 610.
Chicago
Gaina, G., Popa (Gruianu), A."Muscular dystrophy: Experimental animal models and therapeutic approaches (Review)". Experimental and Therapeutic Medicine 21, no. 6 (2021): 610. https://doi.org/10.3892/etm.2021.10042