Retroviral-mediated transfer of the galactocerebrosidase gene in neural progenitor cells
E Torchiana, L Lulli, E Cattaneo, F Invernizzi… - …, 1998 - journals.lww.com
GLOBOID cell leukodystrophy (GCL or Krabbe disease) is a recessive disease caused by
mutations of the lysosomal enzyme galactocerebrosidase (GALC) and twitcher is the murine
model of GCL. We have prepared retroviral packaging cell lines to transduce the GALC
gene. Retroviral transduction restored GALC activity in GCL fibroblasts and increased such
activity to very high levels in immortalized neural progenitor cells (ST14A cells). GALC
activity was also normalized in twitcher fibroblasts co-cultured with ST14A cells over …
mutations of the lysosomal enzyme galactocerebrosidase (GALC) and twitcher is the murine
model of GCL. We have prepared retroviral packaging cell lines to transduce the GALC
gene. Retroviral transduction restored GALC activity in GCL fibroblasts and increased such
activity to very high levels in immortalized neural progenitor cells (ST14A cells). GALC
activity was also normalized in twitcher fibroblasts co-cultured with ST14A cells over …