Article
Retroviral vector-mediated transfer of the galactocerebrosidase (GALC) cDNA leads to overexpression and transfer of GALC activity to neighboring cells.
Biochemical and molecular medicine - 1 Aug 1996
Rafi M A, Fugaro J, Amini S, Luzi P, de Gala G, Victoria T, Dubell C, Shahinfar M, Wenger D A
Abstract excerpt
Galactocerebrosidase (GALC) is responsible for the lysosomal catabolism of certain galactolipids, including galactosylceramide and psychosine. Patients with GALC deficiency have an autosomal recessive disorder known as globoid cell leukodystrophy (GLD) or Krabbe disease. Storage of undegraded glycolipids results in defective myelin and the characteristic globoid cells observed on pathological examination of the...
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