Why the study?
There are no known cures for congenital myopathies, and evaluating the potentials and limitations of mouse models is relevant for translating advances in gene therapy to human applications.
This review provides a comprehensive overview of transgenic mouse models for muscular dystrophies and congenital myopathies, highlighting their translational potential and limitations.
Clinicians should interpret mouse model data cautiously for myopathy therapies; leaves open refinement of preclinical models for better human translation.
Muscular dystrophies are a group of more than 160 different human neuromuscular disorders characterized by a progressive deterioration of muscle mass and strength. The causes, symptoms, age of onset, severity, and progression vary depending on the exact time point of diagnosis and the entity. Congenital myopathies are rare muscle diseases mostly present at birth that result from genetic defects. There are no known cures for congenital myopathies; however, recent advances in gene therapy are promising tools in providing treatment. This review gives an overview of the mouse models used to investigate the most common muscular dystrophies and congenital myopathies with emphasis on their potentials and limitations in respect to human applications.
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Sztretye et al. (2020) studied this question.
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