GATA2 deficiency: flesh and blood.

نویسنده

  • Marshall S Horwitz
چکیده

http://bloodjournal.hematologylibrary.org/content/123/6/799.full.html Updated information and services can be found at: (2182 articles) Free Research Articles • Articles on similar topics can be found in the following Blood collections http://bloodjournal.hematologylibrary.org/site/misc/rights.xhtml#repub_requests Information about reproducing this article in parts or in its entirety may be found online at: http://bloodjournal.hematologylibrary.org/site/misc/rights.xhtml#reprints Information about ordering reprints may be found online at: http://bloodjournal.hematologylibrary.org/site/subscriptions/index.xhtml Information about subscriptions and ASH membership may be found online at:

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GATA2 deficiency-associated bone marrow disorder differs from idiopathic aplastic anemia.

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GATA2 deficiency: a protean disorder of hematopoiesis, lymphatics, and immunity.

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Loss-of-function germline GATA2 mutations in patients with MDS/AML or MonoMAC syndrome and primary lymphedema reveal a key role for GATA2 in the lymphatic vasculature.

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GATA2 haploinsufficiency caused by mutations in a conserved intronic element leads to MonoMAC syndrome.

Previous reports of GATA2 mutations have focused on the coding region of the gene or full gene deletions. We recently identified 2 patients with novel insertion/deletion mutations predicted to result in mRNA nonsense-mediated decay, suggesting haploinsufficiency as the mechanism of GATA2 deficient disease. We therefore screened patients without identified exonic lesions for mutations within con...

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عنوان ژورنال:
  • Blood

دوره 123 6  شماره 

صفحات  -

تاریخ انتشار 2014