The CXCL12–CXCR4 pathway may be a potential target for the treatment of Duchenne muscular dystrophy (DMD), according to a new study published in Bioengineered.

CXCL12 (C-X-C motif chemokine 12), also known as stromal cell-derived factor 1 (SDF1), is a chemokine protein that is ubiquitously expressed in many tissues and cell types. It works by binding to its receptor, CXCR4 (C–X–C motif chemokine receptor 4).

Here, 2 researchers from Nanchang University in China identified CXCL12 as a differentially expressed gene between people with DMD and healthy controls that is also a target of glucocorticoids, one of the main treatments for DMD.

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They constructed a protein-protein interaction network, identified hub genes using bioinformatics tools and found that enriched pathways involving the differentially expressed genes between DMD patients and controls, including CXCL12, were associated with immune response and inflammation.

Read more about DMD etiology

The researchers then conducted experiments using the mdx mouse, a popular model for studying DMD, and found that the levels of both CXCL12 and CXCR4 were increased in these animals. Interestingly, prednisone treatment, which is commonly used to treat patients with DMD, significantly reduced these levels. 

The researchers also showed that metformin, a drug used to treat type 2 diabetes that has been shown to have benefits on dystrophic muscle in mdx mice, reduced the expression of  CXCL12 and CXCR4 in these animals.

“In conclusion, the CXCL12–CXCR4 pathway may be a potential target for DMD therapy,” the researchers wrote. They added that more research is needed to understand the biological functions of these proteins.

Glucocorticoids remain one of the main treatments for DMD, even though they can cause serious side effects. The exact mechanism of action of glucocorticoids and how they benefit patients with DMD is still unclear.


Lai X, Chen J. C-X-C motif chemokine ligand 12: a potential therapeutic target in Duchenne muscular dystrophy. Bioengineered. 2021;12(1):5428-5439. doi:10.1080/21655979.2021.1967029