VEGF ameliorates the ataxic phenotype in spinocerebellar ataxia type 1 (SCA1) mice

SCA1 is an adult-onset, dominantly inherited neurodegenerative disease caused by expansion of a glutamine repeat tract in ATXN1. Although the precise function of ATXN1 remains elusive, it appears to play a role in transcriptional repression. We find that mutant ATXN1 suppresses transcription of the...

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Autores principales: Cvetanovic, Marija, Patel, Jay, Marti, Hugo H, Kini, Ameet R, Opal, Puneet
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2011
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3287040/
https://www.ncbi.nlm.nih.gov/pubmed/22001907
http://dx.doi.org/10.1038/nm.2494
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author Cvetanovic, Marija
Patel, Jay
Marti, Hugo H
Kini, Ameet R
Opal, Puneet
author_facet Cvetanovic, Marija
Patel, Jay
Marti, Hugo H
Kini, Ameet R
Opal, Puneet
author_sort Cvetanovic, Marija
collection PubMed
description SCA1 is an adult-onset, dominantly inherited neurodegenerative disease caused by expansion of a glutamine repeat tract in ATXN1. Although the precise function of ATXN1 remains elusive, it appears to play a role in transcriptional repression. We find that mutant ATXN1 suppresses transcription of the neurotrophic and angiogenic factor VEGF. We also show that genetic or pharmacologic replenishment of VEGF mitigates SCA1 pathogenesis, suggesting a novel therapeutic strategy for this incurable disease.
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spelling pubmed-32870402012-04-16 VEGF ameliorates the ataxic phenotype in spinocerebellar ataxia type 1 (SCA1) mice Cvetanovic, Marija Patel, Jay Marti, Hugo H Kini, Ameet R Opal, Puneet Nat Med Article SCA1 is an adult-onset, dominantly inherited neurodegenerative disease caused by expansion of a glutamine repeat tract in ATXN1. Although the precise function of ATXN1 remains elusive, it appears to play a role in transcriptional repression. We find that mutant ATXN1 suppresses transcription of the neurotrophic and angiogenic factor VEGF. We also show that genetic or pharmacologic replenishment of VEGF mitigates SCA1 pathogenesis, suggesting a novel therapeutic strategy for this incurable disease. 2011-10-16 /pmc/articles/PMC3287040/ /pubmed/22001907 http://dx.doi.org/10.1038/nm.2494 Text en Users may view, print, copy, download and text and data- mine the content in such documents, for the purposes of academic research, subject always to the full Conditions of use: http://www.nature.com/authors/editorial_policies/license.html#terms
spellingShingle Article
Cvetanovic, Marija
Patel, Jay
Marti, Hugo H
Kini, Ameet R
Opal, Puneet
VEGF ameliorates the ataxic phenotype in spinocerebellar ataxia type 1 (SCA1) mice
title VEGF ameliorates the ataxic phenotype in spinocerebellar ataxia type 1 (SCA1) mice
title_full VEGF ameliorates the ataxic phenotype in spinocerebellar ataxia type 1 (SCA1) mice
title_fullStr VEGF ameliorates the ataxic phenotype in spinocerebellar ataxia type 1 (SCA1) mice
title_full_unstemmed VEGF ameliorates the ataxic phenotype in spinocerebellar ataxia type 1 (SCA1) mice
title_short VEGF ameliorates the ataxic phenotype in spinocerebellar ataxia type 1 (SCA1) mice
title_sort vegf ameliorates the ataxic phenotype in spinocerebellar ataxia type 1 (sca1) mice
topic Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3287040/
https://www.ncbi.nlm.nih.gov/pubmed/22001907
http://dx.doi.org/10.1038/nm.2494
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