GALC and lysosomal storage disease: Thus, to determine whether iSC reprogramming could represent a useful platform to model PNS pathology in vitro, we focused on the well-characterized Twitcher (Twi) mouse model30, 31, which carries a spontaneous mutation inactivating the beta-Galactocerebrosidase (GALC) gene function and closely recapitulates the disease manifestations of the human globoid cell leukodystrophy (GLD, or Krabbe's disease), a severe neurodegenerative lysosomal storage disorder.