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Unusual clinical expression and long survival of a pseudouridylate synthase (PUS1) mutation into adulthood

A homozygote missense mutation of the pseudouridylate synthase gene was found in an adult patient with chronic sideroblastic anemia, diarrhea, microcephaly and failure to thrive. Moderate muscle weakness occurred in adulthood (6-min walk distance at 26 years: 240 m, control range 380–782 m) but a pr...

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Bibliografische gegevens
Gepubliceerd in:Eur J Hum Genet
Hoofdauteurs: Metodiev, Metodi D, Assouline, Zahra, Landrieu, Pierre, Chretien, Dominique, Bader-Meunier, Brigitte, Guitton, Corinne, Munnich, Arnold, Rötig, Agnès
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: Nature Publishing Group 2015
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Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4795040/
https://ncbi.nlm.nih.gov/pubmed/25227147
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/ejhg.2014.192
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