Published June 6, 2019 - More info
Dystrophin deficiency leads to progressive muscle degeneration in Duchenne muscular dystrophy (DMD) patients. No known cure exists, and standard care relies on the use of antiinflammatory steroids, which are associated with side effects that complicate long-term use. Here, we report that a single intravenous dose of clinical-stage cardiac stromal cells, called cardiosphere-derived cells (CDCs), improves the dystrophic phenotype in mdx mice. CDCs augment cardiac and skeletal muscle function, partially reverse established heart damage, and boost the regenerative capacity of skeletal muscle. We further demonstrate that CDCs work by secreting exosomes, which normalize gene expression at the transcriptome level, and alter cell signaling and biological processes in mdx hearts and skeletal muscle. The work reported here motivated the ongoing HOPE-2 clinical trial of systemic CDC delivery to DMD patients, and identifies exosomes as next-generation cell-free therapeutic candidates for DMD.
Russell G. Rogers, Mario Fournier, Lizbeth Sanchez, Ahmed G. Ibrahim, Mark A. Aminzadeh, Michael I. Lewis, Eduardo Marbán
Original citation: JCI Insight. 2019;4(7):e125754. https://doi.org/10.1172/jci.insight.125754
Citation for this corrigendum: JCI Insight. 2019;4(11):e130202. https://doi.org/10.1172/jci.insight.130202
Eduardo Marbán’s conflict-of-interest statement was not included in the manuscript. The authors apologize for the oversight. The correct statement is below.
EM declares ownership of Capricor Therapeutics stock (self and spouse) and salary to his spouse from Capricor Therapeutics. He is also the inventor on one awarded (US 9,828,603) and two relevant pending (US 15/516,658 and PCT/US2018/028148) patent applications assigned to Cedars-Sinai Medical Center.
The authors regret the error.
See the related article at Disease-modifying bioactivity of intravenous cardiosphere-derived cells and exosomes in mdx mice.