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J Clin Med. 2012 Jan 19;1(1):1-14. doi: 10.3390/jcm1010001.

Bone Marrow Transplantation Alters the Tremor Phenotype in the Murine Model of Globoid-Cell Leukodystrophy.

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1
Department of Internal Medicine, Campus box 8007, 660 South Euclid Avenue, Washington University School of Medicine, St. Louis, MO 63110, USA.

Abstract

Tremor is a prominent phenotype of the twitcher mouse, an authentic genetic model of Globoid-Cell Leukodystrophy (GLD, Krabbe's disease). In the current study, the tremor was quantified using a force-plate actometer designed to accommodate low-weight mice. The actometer records the force oscillations caused by a mouse's movements, and the rhythmic structure of the force variations can be revealed. Results showed that twitcher mice had significantly increased power across a broad band of higher frequencies compared to wildtype mice. Bone marrow transplantation (BMT), the only available therapy for GLD, worsened the tremor in the twitcher mice and induced a measureable alteration of movement phenotype in the wildtype mice. These data highlight the damaging effects of conditioning radiation and BMT in the neonatal period. The behavioral methodology used herein provides a quantitative approach for assessing the efficacy of potential therapeutic interventions for Krabbe's disease.

KEYWORDS:

bone marrow transplantation; galactosylceramidase; globoid-cell leukodystrophy; lysosomal storage disease; tremor

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