Key result
Systemic AAV9-DWORF gene therapy reduces myocardial fibrosis and improves heart hemodynamics in aged mdx mice.
Why the study?
Does adeno-associated virus serotype 9-DWORF therapy improve cardiac function and exercise capacity in aged mdx mice?
Does adeno-associated virus serotype 9-DWORF therapy improve cardiac function and exercise capacity in aged mdx mice?
DWORF gene therapy reduces myocardial fibrosis and improves cardiac function and exercise capacity in a mouse model of Duchenne muscular dystrophy cardiomyopathy.
Hypothesis-generating for DWORF gene therapy in DMD cardiomyopathy; requires human trials before clinical consideration.
vector genome particles/mouse) via the tail vein. Exercise capacity, heart histology, and cardiac function were examined at 18 months of age. We found DWORF expression was significantly reduced at the transcript and protein levels in mdx mice. Adeno-associated virus serotype 9-DWORF vector significantly enhanced SERCA activity. Systemic adeno-associated virus serotype 9-DWORF therapy reduced myocardial fibrosis and improved treadmill running, electrocardiography, and heart hemodynamics. Conclusions Our data suggest that DWORF deficiency contributes to SERCA dysfunction in mdx mice and that DWORF gene therapy holds promise to treat DMD cardiomyopathy.
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Morales et al. (2023) studied Duchenne Muscular Dystrophy Cardiomyopathy. Adeno-associated virus serotype 9-DWORF vector was evaluated on Exercise capacity, heart histology, and cardiac function. Systemic adeno-associated virus serotype 9-DWORF gene therapy reduced myocardial fibrosis and improved treadmill running, electrocardiography, and heart hemodynamics in aged mdx mice.
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