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Reversal of cardiac and skeletal manifestations of Duchenne muscular dystrophy by cardiosphere-derived cells and their exosomes in mdx dystrophic mice and in human Duchenne cardiomyocytes

Mark A. Aminzadeh, Russell G. Rogers, Kenneth Gouin, Mario Fournier, Rachel E. Tobin, Xuan Guan, Martin K. Childers, Allen M. Andres, David J. Taylor, Ahmed Ibrahim, Xiangming Ding, Angelo Torrente, Joshua M. Goldhaber, Ronald A. Victor, Roberta A. Gottlieb, Michael Lewis, Eduardo Marbán
doi: https://doi.org/10.1101/128900
Mark A. Aminzadeh
Cedars-Sinai Heart Institute, Los Angeles, CA USA;
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Russell G. Rogers
Cedars-Sinai Heart Institute, Los Angeles, CA USA;
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Kenneth Gouin
Cedars-Sinai Heart Institute, Los Angeles, CA USA;
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Mario Fournier
Cedars-Sinai Heart Institute, Los Angeles, CA USA;
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Rachel E. Tobin
Cedars-Sinai Heart Institute, Los Angeles, CA USA;
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Xuan Guan
Institute for Stem Cell and Regenerative Medicine, University of Washington, Seattle, WA USA;
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Martin K. Childers
Institute for Stem Cell and Regenerative Medicine, University of Washington, Seattle, WA USA;
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Allen M. Andres
Cedars-Sinai Heart Institute, Los Angeles, CA USA;
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David J. Taylor
Cedars-Sinai Heart Institute, Los Angeles, CA USA;
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Ahmed Ibrahim
Cedars-Sinai Heart Institute, Los Angeles, CA USA;
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Xiangming Ding
UCLA Technology Center for Genomics & Bioinformatics, Los Angeles, CA USA
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Angelo Torrente
Cedars-Sinai Heart Institute, Los Angeles, CA USA;
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Joshua M. Goldhaber
Cedars-Sinai Heart Institute, Los Angeles, CA USA;
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Ronald A. Victor
Cedars-Sinai Heart Institute, Los Angeles, CA USA;
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Roberta A. Gottlieb
Cedars-Sinai Heart Institute, Los Angeles, CA USA;
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Michael Lewis
Cedars-Sinai Heart Institute, Los Angeles, CA USA;
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Eduardo Marbán
Cedars-Sinai Heart Institute, Los Angeles, CA USA;
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Abstract

Genetic deficiency of dystrophin leads to disability and premature death in Duchenne muscular dystrophy, affecting the heart as well as skeletal muscle. Here we report that cardiosphere-derived cells (CDCs), which are being tested clinically for the treatment of Duchenne cardiomyopathy, improve cardiac and skeletal myopathy in the mdx mouse model of DMD and in human Duchenne cardiomyocytes. Injection of CDCs into the hearts of mdx mice augments cardiac function, ambulatory capacity and survival. Exosomes secreted by human CDCs reproduce the benefits of CDCs in mdx mice and in human Duchenne cardiomyocytes. The findings further motivate the testing of CDCs in Duchenne patients, while identifying exosomes as next-generation therapeutic candidates.

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Posted April 20, 2017.
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Reversal of cardiac and skeletal manifestations of Duchenne muscular dystrophy by cardiosphere-derived cells and their exosomes in mdx dystrophic mice and in human Duchenne cardiomyocytes
Mark A. Aminzadeh, Russell G. Rogers, Kenneth Gouin, Mario Fournier, Rachel E. Tobin, Xuan Guan, Martin K. Childers, Allen M. Andres, David J. Taylor, Ahmed Ibrahim, Xiangming Ding, Angelo Torrente, Joshua M. Goldhaber, Ronald A. Victor, Roberta A. Gottlieb, Michael Lewis, Eduardo Marbán
bioRxiv 128900; doi: https://doi.org/10.1101/128900
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Reversal of cardiac and skeletal manifestations of Duchenne muscular dystrophy by cardiosphere-derived cells and their exosomes in mdx dystrophic mice and in human Duchenne cardiomyocytes
Mark A. Aminzadeh, Russell G. Rogers, Kenneth Gouin, Mario Fournier, Rachel E. Tobin, Xuan Guan, Martin K. Childers, Allen M. Andres, David J. Taylor, Ahmed Ibrahim, Xiangming Ding, Angelo Torrente, Joshua M. Goldhaber, Ronald A. Victor, Roberta A. Gottlieb, Michael Lewis, Eduardo Marbán
bioRxiv 128900; doi: https://doi.org/10.1101/128900

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