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Single SERCA2a Therapy Ameliorated Dilated Cardiomyopathy for 18 Months in a Mouse Model of Duchenne Muscular Dystrophy

Molecular Therapy (2020) 28(3) 845-854
DOI: 10.1016/j.ymthe.2019.12.011
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Abstract

Increased cytosolic calcium level is a pathogenic hallmark in Duchenne muscular dystrophy. The sarcoplasmic/endoplasmic reticulum (SR) calcium ATPase (SERCA) pump transfers cytosolic calcium to the SR. Wasala et al. show that AAV-mediated overexpression of SERCA2a at 3 months of age resulted in lifelong prevention of cardiomyopathy in a mouse model.

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Wasala, N. B., Yue, Y., Lostal, W., Wasala, L. P., Niranjan, N., Hajjar, R. J., … Duan, D. (2020). Single SERCA2a Therapy Ameliorated Dilated Cardiomyopathy for 18 Months in a Mouse Model of Duchenne Muscular Dystrophy. Molecular Therapy, 28(3), 845–854. https://doi.org/10.1016/j.ymthe.2019.12.011

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