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

Marija Cvetanovic, Jay M. Patel, Hugo H. Marti, Ameet R. Kini, Puneet Opal*

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

69 Scopus citations

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.

Original languageEnglish (US)
Pages (from-to)1445-1447
Number of pages3
JournalNature Medicine
Volume17
Issue number11
DOIs
StatePublished - Nov 2011

ASJC Scopus subject areas

  • Biochemistry, Genetics and Molecular Biology(all)

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