Why the study?
Does skeletal muscle-specific micro-dystrophin expression improve dystrophic heart disease in aged mdx mice?
Population
Aged mdx mice (23 months of age), a model of Duchenne cardiomyopathy
Comparison
Skeletal muscle-specific micro-dystrophin… vs Non-transgenic original mdx mice
Design
Preclinical
Follow-up
Aged to 23 months
Authors
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Does not support skeletal muscle-only therapy for cardiac DMD; leaves open dual skeletal-cardiac targeting in future gene therapy studies.
Does skeletal muscle-specific micro-dystrophin expression improve dystrophic heart disease in aged mdx mice?
Exclusive skeletal muscle correction does not modulate dystrophic heart disease in the aged mdx model, underscoring the importance of targeting both skeletal and cardiac muscles in DMD therapy.
Wasala et al. (2013) studied this question.
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