Exome sequencing reveals a thrombopoietin ligand mutation in a Micronesian family with autosomal recessive aplastic anemia

  • Majed J. Dasouki
    Department of Pediatrics,
  • Syed K. Rafi
    Department of Anatomy and Cell Biology, University of Kansas Medical Center, Kansas City, KS;
  • Adam J. Olm-Shipman
    Department of Anatomy and Cell Biology, University of Kansas Medical Center, Kansas City, KS;
  • Nathan R. Wilson
    Department of Anatomy and Cell Biology, University of Kansas Medical Center, Kansas City, KS;
  • Sunil Abhyankar
    Department of Internal Medicine, and
  • Brigitte Ganter
    DNAnexus, Mountain View, CA;
  • L. Mike Furness
    DNAnexus, Mountain View, CA;
  • Jianwen Fang
    Applied Bioinformatics Laboratory, University of Kansas, Lawrence, KS; and
  • Rodrigo T. Calado
    Department of Internal Medicine, University of São Paulo at Ribeirão Preto Medical School, Ribeirão Preto, São Paulo, Brazil
  • Irfan Saadi
    Department of Anatomy and Cell Biology, University of Kansas Medical Center, Kansas City, KS;

書誌事項

公開日
2013-11-14
DOI
  • 10.1182/blood-2012-12-473538
公開者
American Society of Hematology

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説明

<jats:title>Key Points</jats:title> <jats:p>Recessive mutations in the thrombopoietin gene are a novel cause of aplastic anemia. Such patients may benefit from treatment with eltrombopag or romiplostim.</jats:p>

収録刊行物

  • Blood

    Blood 122 (20), 3440-3449, 2013-11-14

    American Society of Hematology

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