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

Andrew M Yeager, Susan Brennan, Carol Tiffany, Hugo W. Moser, George W. Santos

Research output: Contribution to journalArticle

117 Citations (Scopus)

Abstract

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.

Original languageEnglish (US)
Pages (from-to)1052-1054
Number of pages3
JournalScience
Volume225
Issue number4666
StatePublished - 1984
Externally publishedYes

Fingerprint

Cell Transplantation
Globoid Cell Leukodystrophy
Survival
Sphingolipidoses
Transplantation
Enzyme Replacement Therapy
Inborn Genetic Diseases
Central Nervous System Diseases
Demyelinating Diseases
Enzymes
Blood-Brain Barrier
Peripheral Nerves
Central Nervous System
Animal Models
Transplants
Therapeutics

ASJC Scopus subject areas

  • General

Cite this

Yeager, A. M., Brennan, S., Tiffany, C., Moser, H. W., & Santos, G. W. (1984). Prolonged survival and remyelination after hematopoietic cell transplantation in the twitcher mouse. Science, 225(4666), 1052-1054.

Prolonged survival and remyelination after hematopoietic cell transplantation in the twitcher mouse. / Yeager, Andrew M; Brennan, Susan; Tiffany, Carol; Moser, Hugo W.; Santos, George W.

In: Science, Vol. 225, No. 4666, 1984, p. 1052-1054.

Research output: Contribution to journalArticle

Yeager, AM, Brennan, S, Tiffany, C, Moser, HW & Santos, GW 1984, 'Prolonged survival and remyelination after hematopoietic cell transplantation in the twitcher mouse', Science, vol. 225, no. 4666, pp. 1052-1054.
Yeager, Andrew M ; Brennan, Susan ; Tiffany, Carol ; Moser, Hugo W. ; Santos, George W. / Prolonged survival and remyelination after hematopoietic cell transplantation in the twitcher mouse. In: Science. 1984 ; Vol. 225, No. 4666. pp. 1052-1054.
@article{a6e6af7792b94c6ab36a71acc7fb0a32,
title = "Prolonged survival and remyelination after hematopoietic cell transplantation in the twitcher mouse",
abstract = "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.",
author = "Yeager, {Andrew M} and Susan Brennan and Carol Tiffany and Moser, {Hugo W.} and Santos, {George W.}",
year = "1984",
language = "English (US)",
volume = "225",
pages = "1052--1054",
journal = "Science",
issn = "0036-8075",
publisher = "American Association for the Advancement of Science",
number = "4666",

}

TY - JOUR

T1 - Prolonged survival and remyelination after hematopoietic cell transplantation in the twitcher mouse

AU - Yeager, Andrew M

AU - Brennan, Susan

AU - Tiffany, Carol

AU - Moser, Hugo W.

AU - Santos, George W.

PY - 1984

Y1 - 1984

N2 - 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.

AB - 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.

UR - http://www.scopus.com/inward/record.url?scp=0021249707&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=0021249707&partnerID=8YFLogxK

M3 - Article

C2 - 6382609

AN - SCOPUS:0021249707

VL - 225

SP - 1052

EP - 1054

JO - Science

JF - Science

SN - 0036-8075

IS - 4666

ER -