GALC and Krabbe disease: Several pre-clinical studies have shown GALC expression and variable clinical–pathological amelioration in the Twitcher (Twi) mouse (a GALC mutant that recapitulates the severe form of GLD) upon hematopoietic (14), neural (15) and mesenchymal (16) stem cell transplant, intracerebral GT using adeno-associated vectors (AAV) (17,18) and lentiviral vectors (LV) (19), or combination of therapies (20–24).