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Nat Med. 2011 Oct 16;17(11):1445-7. doi: 10.1038/nm.2494.

Vascular endothelial growth factor ameliorates the ataxic phenotype in a mouse model of spinocerebellar ataxia type 1.

Author information

  • 1Davee Department of Neurology, Northwestern University Feinberg School of Medicine, Chicago, Illinois, USA.

Abstract

Spinocerebellar ataxia type 1 (SCA1) is an adult-onset, dominantly inherited neurodegenerative disease caused by expansion of a glutamine repeat tract in ataxin-1 (ATXN1). Although the precise function of ATXN1 remains elusive, it seems to be involved in transcriptional repression. We find that mutant ATXN1 represses transcription of the neurotrophic and angiogenic factor vascular endothelial growth factor (VEGF). Genetic overexpression or pharmacologic infusion of recombinant VEGF mitigates SCA1 pathogenesis, suggesting a new therapeutic strategy for this disease.

PMID:
22001907
[PubMed - indexed for MEDLINE]
PMCID:
PMC3287040
Free PMC Article

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