Retroviral-mediated transfer of the galactocerebrosidase gene in neural progenitor cells

Elena Torchiana, Laura Lulli, Elena Cattaneo, Federica Invernizzi, Rita Orefice, Barbara Bertagnolio, Stefano Di Donato, Gaetano Finocchiaro

Research output: Contribution to journalArticlepeer-review

Abstract

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-expressing GALC, demonstrating that this enzyme is secreted and can be imported efficiently by GALC-deficient cells. These results give the necessary background to evaluate the therapeutic effect in twitcher of brain grafting of neural progenitor cells engineered to release high levels of GALC.

Original languageEnglish
Pages (from-to)3823-3827
Number of pages5
JournalNeuroReport
Volume9
Issue number17
Publication statusPublished - Dec 1 1998

Keywords

  • Gene therapy
  • Krabbe disease
  • Neural progenitor cells
  • Retrovirus

ASJC Scopus subject areas

  • Neuroscience(all)

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