Anti-ADAMTS1 antibody relieved muscle dysfunction and fibrosis in a mouse model with Duchenne muscular dystrophy (DMD), found in a new study by Chinese researchers published in the journal Life Sciences. This finding suggests ADAMTS1 could be a new therapeutic target for the disease.

ADAMTS1 is a metalloproteinase that was reported to be highly expressed in mdx mice, the most common animal model of DMD. It binds to extracellular matrix proteins and contributes to cell-cell and cell-extracellular matrix interactions.

The study aimed to elucidate the role of ADAMTS1 in muscle function, fibrosis and damage, and respiratory function. Researchers from Xi’an Children’s Hospital in Shaanxi Province, China led by Dong Wang, MD, used multiplex ligation-dependent probe amplification assay and next-generation sequencing. These methods were used to genetically diagnose 102 DMD patients and their mothers. They also treated mdx mice with anti-ADAMTS1 antibody for 3 weeks.

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Researchers found that ADAMTS1 levels were increased in the serum of patients with DMD as well as in the gastrocnemius muscle of the mdx mice.

Anti-ADAMTS1 antibody treatment increased the transcription of versican, a large extracellular matrix proteoglycan, but decreased protein expression. Moreover, anti-ADAMTS1 treatment improved muscle strength, the function of the diaphragm, and the extensor digitorum longus muscles in the animals. It also relieved muscle dysfunction and fibrosis.

“ADAMTS1 may serve as an encouraging target for developing new biological therapies for DMD,” the researchers concluded.

DMD is caused by mutations in the DMD gene, which leads to cells not being able to synthesize any functional dystrophin protein. Dystrophin is essential for the health of muscles cells and in its absence, fibrous tissue begins to form. Fibrosis is characterized by the excessive deposition of extracellular matrix proteins and directly induces progressive muscle dysfunction, the authors said. There are currently no effective treatments that can suppress muscle fibrosis in DMD.


Wang Y, Xiao Y, Zheng Y, Yang L, Wang D. An anti-ADAMTS1 treatment relieved muscle dysfunction and fibrosis in dystrophic mice. Life Sci. 2021;15;281:119756. doi:10.1016/j.lfs.2021.119756