Krabbe leukodystrophy (KD) is a progressive and fatal neurologic lysosomal storage disorder that usually affects infants and causes death before two to three years of age. Hematopoietic Stem Cell Therapy extends the long-term survival with improved quality of life for KD patients, but it is not a cure. Using our newly developed KD animal model, we will identify which brain cell(s) needs to be efficiently cured with therapy.

Project financed by ELA up to:  71 082 €