Spinocerebellar ataxia type 1 (SCA1) is caused by a CAG repeat expansion in the ataxin-1 (ATXN1) gene. Experiments in a mouse model of SCA1 have now shown that the mutant ATXN1 protein represses transcription of the gene that encodes vascular endothelial growth factor (VEGF), and that overexpression or infusion of VEGF improves motor function in this animals. The authors propose that VEGF could represent a novel therapeutic strategy for SCA1.
ORIGINAL RESEARCH PAPER
Cvetanovic, M. et al. Vascular endothelial growth factor ameliorates the ataxic phenotype in a mouse model of spinocerebellar ataxia type 1. Nat. Med. 17, 1445–1447 (2011)
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Vascular endothelial growth factor alleviates spinocerebellar ataxia type 1 in mice. Nat Rev Neurol 8, 3 (2012). https://doi.org/10.1038/nrneurol.2011.197
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DOI: https://doi.org/10.1038/nrneurol.2011.197
