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Could Cell Therapy Boost Cardiac Function in DMD Patients?

Jan 14, 2020 10:06:00 AM / by Stacy Matthews Branch, DVM, PhD

Push to overcome obstacles_AdobeStock_112997034-1Mesenchymal Stem cells (MSCs) derived from mouse muscle tissue and bone marrow were used in an experimental cell therapy for Duchenne Muscular Dystrophy.

Duchenne muscular dystrophy (DMD) is an incurable, progressively debilitating muscular-skeletal and cardiac disease caused by a mutation in the dystrophin gene. The encoded dystrophin protein is part of a larger protein complex involved in anchoring the muscle cytoskeleton to components of the extracellular matrix. Loss of dystrophin leads to muscle wasting, and heart disease is a major cause of death in patients with DMD. Therefore, the availability of effective treatments to address cardiac function in people with DMD is vital.

A collaboration of scientists from Poznan University of Medical Sciences and the University of Illinois conducted studies to restore dystrophin by transplanting dystrophin expressing chimeric cells (DECs) in a mouse model of DMD. Results of the team’s earlier research showed that myoblast-derived DECs transplanted into a DMD mouse model showed increased dystrophin levels, a decrease in an inflammatory response, and marked improvement in cardiac muscle function. The researchers later conducted studies to determine the effects of interosseous injection of myoblast- and mesenchymal stem cell-derived DECs in DMD mice.

Myoblasts and mesenchymal stem cells (MSCs) were derived from mouse muscle tissue and bone marrow, respectively. DECs were created using these cells via a fusion procedure and then transplanted into the mice via interosseous injection. By 90 days post-injection, immunofluorescence analysis showed an increase in dystrophin expression in muscle tissue. There was also a decrease in cardiac muscle fibrosis when compared to vehicle-injected (control) DMD mice. Echocardiography showed improved heart function in DEC-injected mice when compared to controls.

DECs may represent a viable approach to the treatment of patients with DMD. The use of mesenchymal stem cell-derived DECs provides beneficial immunomodulatory features. The cardioprotective role of DECs can help increase survival time and quality of life for those with DMD, particularly given the fact that cardiac disease is one of the main causes of death in this patient population.

Learn more about the capabilities of mesenchymal stem cells (MSCs) derived from bone marrow aspirate and HemaCare’s recently launched GMPrimeTM Bone Marrow Aspirate.

Resource: Experimental Cell Therapy Improves Cardiac Function in Mouse Model of DMD, Study Says. (2019). Muscular Dystrophy News. Retrieved 2 December 2019, from

Topics: Bone Marrow, Cell Therapy, Stem Cells

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