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Congenital erythropoietic porphyria due to a mutation in GATA1: the first trans-acting mutation causative for a human porphyria

Congenital erythropoietic porphyria (CEP), an autosomal recessive disorder, is due to mutations of uroporphyrinogen III synthase (UROS). Deficiency of UROS results in excess uroporphyrin I, which causes photosensitization. We evaluated a 3-year-old boy with CEP. A hypochromic, microcytic anemia was...

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Autors principals: Phillips, John D., Steensma, David P., Pulsipher, Michael A., Spangrude, Gerald J., Kushner, James P.
Format: Artigo
Idioma:Inglês
Publicat: American Society of Hematology 2007
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Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC1852202/
https://ncbi.nlm.nih.gov/pubmed/17148589
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2006-06-022848
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