In summary, there is a molecule which blocks the Nrg1TIII in CMT1B, causing the demyelination of the nerves. Both papers found that if the strength or amount of Nrg1TIII can be controlled, the nerves affected by CMT may be restored. When more of the protein is added to lab models with demyelinating types of CMT, the damaged nerves are partially repaired.
These papers proved that if the molecule that blocks Nrg1TIII activity can be suppressed, the neuropathy is improved. Therefore, stimulating Nrg1TIII by targeting the molecule that blocks the protein could be a great treatment for demyelinating CMTs. Basically, inhibiting the inhibitor of Nrg1TIII seems to repair the nerves without a toxic overload.
These papers were published in tandem by Dr. Maurizio D’Antonio, Larry Wrabtez and Laura Feltri. The CMT Research Foundation is in active discussions with the researchers to determine how we can collectively advance this breakthrough.
Read the summaries of the papers: https://academic.oup.com/hmg/advance-article-abstract/doi/10.1093/hmg/ddy411/5210922
