Journal
EXPERIMENTAL AND THERAPEUTIC MEDICINE
Volume 21, Issue 6, Pages -Publisher
SPANDIDOS PUBL LTD
DOI: 10.3892/etm.2021.10042
Keywords
muscular dystrophy; animal models; gene therapy; mdx mouse; canine model; pig model; read-through; exon skipping
Categories
Funding
- Ministry of Research and Innovation in Romania, under Program 1-The Improvement of the National System of Research and Development, Subprogram 1.2-Institutional ExcellenceProjects of Excellence Funding in RDI [7PFE/16.10.2018]
- [31N/2016/PN 16.22.02.05]
- [1N/2019/PN19.29.01.03]
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Muscular dystrophies are a group of genetically inherited diseases characterized by muscle weakness and wasting, with no effective treatment currently available. In recent years, several novel therapeutic approaches have been developed, including restoring protein expression and genetically engineered animal models.
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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