A promising new study on CMT2A published on the Journal of Clinical Investigation

Schermata 2022-11-09 alle 15.13.48

According to a new study on a mouse model, blocking the activity of a protein known as SARM1 may be a useful strategy in Charcot-Marie-Tooth disease type 2A (CMT2A). A group of St. Louis researchers argue that such a strategy can prevent nerve loss and other neurodegenerative disorders.

“Our latest research suggests that […] we may be able to interfere with SARM1 ‘s ability to trigger harmful neuroinflammation” said Jeffrey Milbrandt, MD, PhD, one of the study’s senior authors and a professor at Washington University School of Medicine, Missouri, in its press release.

“We desperately need treatments for neurodegenerative diseases ,” Milbrandt said. “ With evidence of the central role of SARM1 in these diseases, we are very interested in finding ways to block this molecule – either with small molecule inhibitors or gene therapy techniques .”

The study A SARM1/mitochondrial feedback loop drives neuropathogenesis in a Charcot-Marie-Tooth disease type 2A rat model was published in the Journal of Clinical Investigation.

We thank the Charcot Marie Tooth News online magazine for highlighting this important news (click on the link to read the whole article) and one of our patients for reporting it to us.

*is a critical mediator of a molecular pathway leading to programmed axon destruction.

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