Correction of the galactocerebrosidase deficiency in globoid cell leukodystrophy-cultured cells by SL3-3 retroviral-mediated gene transfer.
Globoid cell leukodystrophy (GCL) or Krabbe disease is an autosomal recessive inherited disease caused by the deficiency of galactocerebrosidase, the lysosomal enzyme responsible for the degradation of galactocerebroside, a major component of myelin. An animal model homologue of GCL is the twitcher mouse. In the present work, using novel recombinant retroviruses harboring the SL3-3 LTR, we have been able to stably correct the galactocerebrosidase deficiency in twitcher mouse TM-2 cells and in primary human fibroblasts from a patient with globoid cell leukodystrophy. These results show the possibility of retroviral-mediated gene therapy for the treatment of GCL.
Gama Sosa
M A
MA
Department of Neurology, New York University School of Medicine, New York 10016, USA.
de Gasperi
R
R
Undevia
S
S
Yeretsian
J
J
Rouse
S C
SC
Lyerla
T A
TA
Kolodny
E H
EH
D000818:Animals; D002460:Cell Line; D002522:Chlorocebus aethiops; D005698:Galactosylceramidase / Q000172:deficiency*; D018014:Gene Transfer Techniques; D015316:Genetic Therapy; D005822:Genetic Vectors; D006801:Humans; D007965:Leukodystrophy, Globoid Cell / Q000201:enzymology* / Q000235:genetics / Q000628:therapy; D051379:Mice
D005698:Galactosylceramidase