Grant Will Further Research of Dental Pulp Stem Cells in CMT1A
A researcher from Belgium who is developing a new cell-based model for Charcot-Marie-Tooth (CMT) disease has been awarded nearly $99,000 to further her research.
Wolfs has been developing a new human stem cell model for CMT1A, the most common subtype of CMT, using stem cells extracted from the pulp of teeth (the center of the tooth that contains blood vessels and nerves), removed during dental procedures.
These dental pulp stem cells (hDPSCs) can differentiate into dental pulp tissue and into other cell types, providing an alternative to induced pluripotent stem cells, which are collected from blood or skin cells.
CMT1A is caused by an extra copy of the PMP22 gene. This gene provides instructions to make a crucial component of the myelin sheath, the fatty covering of nerve extensions (called axons) that transmit nerve signals, produced by Schwann cells in the peripheral nervous system — outside the brain and the spinal cord. However, there currently are no human stem cell models that accurately depict the Schwann cells from CMT1A patients.
The first goal of this project is to ensure that hDPSCs indeed can form myelin, and the second goal is to investigate whether these cells can express (activate) the CMT1A markers found in the Schwann cells of people with CMT1A.
If successful, this model may provide insight into the signaling pathways involved in CMT1A development, and potentially be translatable to other CMT1 subtypes that also occur due to a genetic mutation in Schwann cells.
“A reliable system that accurately reports the function in the affected Schwann cell will directly facilitate the identification of potential new drug targets and speed the design of new therapeutics,” the CMTA stated.
Through its STAR banner, launched in 2008 to fund research in CMT and quicken the development of new treatments, the CMTA has invested more than $17 million, with plans to invest another $10 million over the next few years. STAR gathers the efforts of a global network of biotech research partners, research scientists, clinicians, and patients.