期刊
JOURNAL OF CLINICAL MEDICINE
卷 1, 期 1, 页码 1-14出版社
MDPI
DOI: 10.3390/jcm1010001
关键词
bone marrow transplantation; lysosomal storage disease; tremor galactosylceramidase; globoid-cell leukodystrophy
资金
- NIH [R01 HD055461, NIH R01 NS043205]
- Hunter's Hope foundation
- National Tay-Sach's and Allied Diseases Association (ASR)
- NIH Neuroscience Blueprint Interdisciplinary Center Core [HD06217110]
- Center Core Grant [HD02528]
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.
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