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The future for treatment by bone marrow transplantation for adrenoleukodystrophy, metachromatic leukodystrophy, globoid cell leukodystrophy and Hurler syndrome

โœ Scribed by W. Krivit; L. A. Lockman; P. A. Watkins; J. Hirsch; E. G. Shapiro

Publisher
Springer
Year
1995
Tongue
English
Weight
995 KB
Volume
18
Category
Article
ISSN
0141-8955

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## Abstract Bone marrow transplantation (BMT) has therapeutic value for twitcher (globoid cell leukodystrophy) mice, which suffer from a genetic deficiency of the lysosomal enzyme galactosylceramidase that leads to progressive demyelination and early death. Preliminary investigations indicated that