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Autologous c-kit+ cardiac stem cells (CSCs) are an experimental cell therapy being investigated for the treatment of congenital heart defects, specifically Hypoplastic Left Heart Syndrome (HLHS). These cells are isolated from the patient's own cardiac tissue (typically obtained during a planned surgical procedure), expanded ex vivo, and then re-injected into the myocardium. The therapy is defined by the expression of the c-kit (CD117) receptor tyrosine kinase, a marker for cardiac progenitor cells. The mechanism of action involves the potential for these cells to promote myocardial regeneration, reduce fibrosis, and improve ventricular function through paracrine signaling and potential differentiation into cardiac lineages. This specific program, often referred to as the CHILD trial, was developed by researchers including Joshua M. Hare at the University of Miami and has been sponsored by Longeveron.
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