RT Journal Article
SR Electronic
T1 Reversal of cardiac and skeletal manifestations of Duchenne muscular dystrophy by cardiosphere-derived cells and their exosomes in <em>mdx</em> dystrophic mice and in human Duchenne cardiomyocytes
JF bioRxiv
FD Cold Spring Harbor Laboratory
SP 128900
DO 10.1101/128900
A1 Mark A. Aminzadeh
A1 Russell G. Rogers
A1 Kenneth Gouin
A1 Mario Fournier
A1 Rachel E. Tobin
A1 Xuan Guan
A1 Martin K. Childers
A1 Allen M. Andres
A1 David J. Taylor
A1 Ahmed Ibrahim
A1 Xiangming Ding
A1 Angelo Torrente
A1 Joshua M. Goldhaber
A1 Ronald A. Victor
A1 Roberta A. Gottlieb
A1 Michael Lewis
A1 Eduardo Marbán
YR 2017
UL http://biorxiv.org/content/early/2017/04/20/128900.abstract
AB 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.