Research ArticleMuscular Dystrophy

Cystamine Suppresses Polyalanine Toxicity in a Mouse Model of Oculopharyngeal Muscular Dystrophy

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Science Translational Medicine  02 Jun 2010:
Vol. 2, Issue 34, pp. 34ra40
DOI: 10.1126/scitranslmed.3000723

Article Information

vol. 2 no. 34 34ra40

DOI: 
https://doi.org/10.1126/scitranslmed.3000723
PubMed: 
20519718
Published By: 
American Association for the Advancement of Science
Print ISSN: 
1946-6234
Online ISSN: 
1946-6242
History: 
  • Received for publication December 11, 2009
  • Accepted for publication May 10, 2010
  • .
Copyright & Usage: 
Copyright © 2010, American Association for the Advancement of Science

Author Information

  1. Janet E. Davies*,
  2. Claudia Rose*,
  3. Sovan Sarkar and
  4. David C. Rubinsztein†
  1. Department of Medical Genetics, University of Cambridge, Cambridge Institute for Medical Research, Addenbrooke’s Hospital, Hills Road, Cambridge CB2 0XY, UK.
  1. †To whom correspondence should be addressed. E-mail: dcr1000{at}hermes.cam.ac.uk

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  • Cystamine Suppresses Polyalanine Toxicity in a Mouse Model of Oculopharyngeal Muscular Dystrophy

    By Janet E. Davies, Claudia Rose, Sovan Sarkar, David C. Rubinsztein

    Science Translational Medicine : 34ra40

    Mice with oculopharyngeal muscular dystrophy, caused by aggregation of a nuclear protein, show improvement when treated with cystamine, suggesting a possible therapeutic approach for patients.

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