@article {Aminzadeh128900, author = {Mark A. Aminzadeh and Russell G. Rogers and Kenneth Gouin and Mario Fournier and Rachel E. Tobin and Xuan Guan and Martin K. Childers and Allen M. Andres and David J. Taylor and Ahmed Ibrahim and Xiangming Ding and Angelo Torrente and Joshua M. Goldhaber and Ronald A. Victor and Roberta A. Gottlieb and Michael Lewis and Eduardo Marb{\'a}n}, title = {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}, elocation-id = {128900}, year = {2017}, doi = {10.1101/128900}, publisher = {Cold Spring Harbor Laboratory}, 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.}, URL = {https://www.biorxiv.org/content/early/2017/04/20/128900}, eprint = {https://www.biorxiv.org/content/early/2017/04/20/128900.full.pdf}, journal = {bioRxiv} }