Cystamine suppresses polyalanine toxicity in a mouse model of oculopharyngeal muscular dystrophy

Science Translational Medicine

Volumn 2, Issue 34, 2010, Pages

  Davies, Janet E ^a   Rose, Claudia ^a   Sarkar, Sovan ^a   Rubinsztein, David C ^a  

^a UNIVERSITY OF CAMBRIDGE   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

ALANINE; CYSTAMINE; POLYADENYLIC ACID BINDING PROTEIN; PROTEIN GLUTAMINE GAMMA GLUTAMYLTRANSFERASE 2; BIOLOGICAL MARKER; PEPTIDE; POLYALANINE; PROTEIN GLUTAMINE GAMMA GLUTAMYLTRANSFERASE;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; APOPTOSIS; ARTICLE; CELL NUCLEUS; CELL STRAIN HEK293; CONTROLLED STUDY; DISEASE MODEL; DRUG TARGETING; EMBRYO; ENZYME ACTIVITY; ENZYME INHIBITION; GENE MUTATION; GENETIC CODE; HUMAN; HUMAN CELL; INHIBITION KINETICS; MOUSE; MUSCLE WEAKNESS; NONHUMAN; OCULOPHARYNGEAL MUSCULAR DYSTROPHY; PATHOGENESIS; PRIORITY JOURNAL; PROTEIN AGGREGATION; PROTEIN EXPRESSION; SKELETAL MUSCLE; TRINUCLEOTIDE REPEAT; ANIMAL; CELL LINE; CELL PROTECTION; DRUG ANTAGONISM; DRUG EFFECT; ENZYMOLOGY; GENE SILENCING; GENETICS; METABOLISM; PATHOLOGY;

ANIMALS; APOPTOSIS; BIOLOGICAL MARKERS; CELL LINE; CYSTAMINE; CYTOPROTECTION; DISEASE MODELS, ANIMAL; GENE KNOCKDOWN TECHNIQUES; HUMANS; MICE; MUSCLE WEAKNESS; MUSCULAR DYSTROPHY, OCULOPHARYNGEAL; PEPTIDES; TRANSGLUTAMINASES; TRINUCLEOTIDE REPEAT EXPANSION;

EID: 77955617537     PISSN: 19466234     EISSN: 19466242     Source Type: Journal    
DOI: 10.1126/scitranslmed.3000723     Document Type: Article

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44. Acknowledgments: We thank O. Sadiq for technical assistance, G. Johnson Voll for TG2 overexpression constructs, E. Wahle for PABPN1 bacterial expression construct, R. O'Connor and A. Marx for rhabdomyosarcoma cell lines, B. Underwood and B. Ravikumar for helpful comments, and the Muscle Tissue Culture Collection for providing control human myoblasts. Funding: This work was funded by the Wellcome Trust (Senior Fellowship to D.C.R.) and the Muscular Dystrophy Campaign, UK. The Muscle Tissue Culture Collection is part of the German network on muscular dystrophies (MD-NET, service structure S1, 01GM0601) funded by the German Ministry of Education and Research (Bonn, Germany). The Muscle Tissue Culture Collection is a partner of EuroBioBank (http://www.eurobiobank.org) and TREATNMD (http://www.treat-nmd.eu). Author contributions: D.C.R. and J.E.D. designed the experiments; J.E.D., C.R., and S.S. performed the experiments and analyzed the data with D.C.R. J.E.D., C.R., and D.C.R. wrote the manuscript. D.C.R. secured funding. Competing interests: The authors declare that they have no competing interests.