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Science 7 September 1984:
Vol. 225. no. 4666, pp. 1052 - 1054
DOI: 10.1126/science.6382609
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Articles

Science, Vol 225, Issue 4666, 1052-1054
Copyright © 1984 by American Association for the Advancement of Science

articles

Prolonged survival and remyelination after hematopoietic cell transplantation in the twitcher mouse

AM Yeager, S Brennan, C Tiffany, HW Moser, and GW Santos

The twitcher mouse is an animal model of galactosylceramidase deficiency (Krabbe's disease), a human sphingolipidosis. The effects of hematopoietic cell transplantation as potential enzyme replacement therapy were examined in the twitcher mouse. Survival in twitcher mice with transplants was significantly prolonged and was associated with gradual repair of demyelination in peripheral nerves. In contrast, there was no improvement in the neurodegenerative process in the central nervous system after transplantation. These observations indicate that cellular transplantation may effectively provide in vivo enzyme replacement for the peripheral manifestations of genetic storage diseases. Strategies to perturb the blood-brain barrier may be necessary for enzyme replacement to be therapeutic in diseases with central nervous system manifestations.


THIS ARTICLE HAS BEEN CITED BY OTHER ARTICLES:
Specific Determination of {beta}-Galactocerebrosidase Activity via Competitive Inhibition of {beta}-Galactosidase.
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Early Use of Drastic Therapy.
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N. Engl. J. Med. 352, 2124-2126
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Donor-derived cells in the central nervous system of twitcher mice after bone marrow transplantation.
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Review Article: The Inherited Neurodegenerative Disorders of Childhood: Clinical Assessment.
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J Child Neurol 2, 82-97
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