By: Grace Pavlath, PhD, Chief Science Officer
The CMT Research Foundation is pleased to announce a new partnership with The Jackson Laboratory that will help us better understand CMT1B and potentially lead to the discovery of treatments. CMT1B is the third most common form of CMT. It’s caused by a defect in the MPZ gene, which prevents the protective coating around our nerve cells (the myelin sheath) from properly functioning.
The Jackson Laboratory will create several mouse models that will be instrumental in learning how gene mutations in MPZ lead to degeneration of the myelin sheath. This is an important project for CMT drug development because these models will add to the supply of “humanized” models in CMT, of which there are very few today. The models will be made to express the human form of the MPZ gene, which gives a higher probability of success for developing and testing gene therapies. Using these “humanized” models, the translation to people with CMT could be easier.
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