With CMTA support of $98,890, researchers at Hasselt University in Belgium, led by Esther Wolfs, PhD, are developing an innovative “patient-in-a-dish” model for CMT1A using dental pulp stem cells (hDPSC). This approach provides a non-invasive way to study Schwann cells, which are critical for understanding the disease mechanisms of CMT1A.
The team collected wisdom teeth from 20 donors representing eight subtypes of CMT through CMTA’s Patients as Partners in Research program. Using these stem cells, they successfully created Schwann cells with an overexpression of PMP22, replicating the genetic mechanisms of CMT1A. This breakthrough allows researchers to study how Schwann cells are affected by the disease and explore potential treatments more quickly and cost-effectively than traditional animal models.
In addition to creating a model for CMT1A, the team is working to develop cell lines for other subtypes, including CMT1E, HNPP, CMTX1 (aka CMT1X or CMTX), CMT2A, CMT2C, CMT2T, and CMT4A. This collaborative effort highlights the power of community involvement and provides an important resource for accelerating therapeutic development for multiple types of CMT.
By transforming how CMT is studied, this project is helping to remove barriers to progress in research and treatments. CMTA’s Strategy To Accelerate Research (CMTA-STAR) collaborative model continues to fuel groundbreaking innovation, bringing hope for treatments and a brighter future for everyone living with CMT.
