Novel stem cell transplantation strategy to treat Duchenne muscular dystrophy

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Novel stem cell transplantation strategy to treat Duchenne muscular dystrophy
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Duchenne muscular dystrophy (DMD) is a common progressive muscular disorder. It is an X-chromosome-linked disorder that gradually weakens various muscle groups including those supporting the heart, thus resulting into early death. While steroids are widely used to regulate severe inflammation in patients with DMD, they are known to cause serious adverse effects.

The team monitored the hAMSC-treated and untreated mdx mice using a variety of tests. These included standard blood tests, histological examinations, spontaneous wheel-running activities, assessment of grip strength, and echocardiography. Treatment with hAMSCs showed significant promise, especially when delivered during the early phase of DMD.

For instance, the proportion of anti-inflammatory M2 macrophages increased significantly in hAMSC-treated mdx mice. Quite notably, mdx mice that received the hAMSC treatment showed a better grip strength than the untreated mdx mice. The treated mdx mice showed a transient downregulation of serum creatine kinase after repeated systemic hAMSC injections."The downregulation of serum creatine kinase is usually associated with the suppression of muscle damage," comments Dr. Nitahara‑Kasahara.

The hAMSC treatment also resulted in the regulation of muscle damage following inflammation. In addition, it improved the histological appearance of the previously impacted skeletal muscles. Quite notably, the hAMSC-treated mdx mice also covered a greater distance per minute than their untreated counterparts during standard activity tests.

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