Charcot – Marie Tooth disease, which gradually leads to paralysis of the legs and hands, is incurable. But this sentence may soon be written in the past tense, thanks to the work of French researchers.
This pathology is an inherited neurological disease. It is also the most widespread in the world. It is caused by the overproduction of a protein, called PMP22, by the body.
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An identified target
However, any protein is made from an RNA sequence, a copy of DNA that contains the necessary information.
The researchers, from the CNRS, Inserm, AP-HP, the universities of Paris and Paris-Saclay, have succeeded in identifying the RNA sequence corresponding to the PMP22 protein. And a way to stop its transmission, thanks to smaller pieces of RNA interfering with the target.
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A “complete restitution” of the movement
The first trials of this therapy, in mice, allowed “the complete and rapid restoration of locomotor activity and strength”, report the authors of this work.
Next steps: preclinical and clinical trials in humans.
Sources: CNRS, Inserm, AP-HP, Universities of Paris and Paris-Saclay, March 2021.
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