Recent insights suggest that in addition to white matter and myelin, nerve cells (also called gray matter) and their processes (axons) are damaged in patients with various white matter disorders. The research of Heine and her colleagues will focus specifically on the involvement of these nerve cells in white matter abnormalities in patients. An innovative approach will be used: modern - OMICS and functional activity measurements will be combined with advanced 2D and 3D iPSC and organoid models.
Pediatric neurologist Dr. Nicole Wolf is closely involved in the research and indicates: "This project represents a huge step forward for patients with 4H leukodystrophy, as there is no animal model for this rare genetic disease."
Check out the other selected projects within the transnational call of EJP RD.