The cellular pathogenesis of paroxysmal nocturnal haemoglobinuria Journal Article

  


Authors: Karadimitris, A.; Luzzatto, L.
Article Title: The cellular pathogenesis of paroxysmal nocturnal haemoglobinuria
Abstract: Paroxysmal nocturnal haemoglobinuria (PNH) is a unique disorder characterised by the triad of intravascular haemolysis, thrombosis and bone marrow failure. In the early seventies it was shown that PNH is a clonal disease; and in the nineties the molecular basis of the PNH abnormality was elucidated. However, what makes a PNH clone expand is still not known. Here, we suggest that this is due to somatic cell selection, resulting from the presence in the patient of autoreactive T cells that target glycosylphosphatidylinositol (GPI) in the context of an MHC-like molecule on the surface of haemopoietic stem cells. PNH cells would escape damage precisely because they have lost most or all of their ability to produce GPI.
Keywords: controlled study; gene mutation; pathogenesis; t lymphocyte; gene targeting; somatic cell; cell protein; editorial; hemoglobin; vein thrombosis; cell damage; lymphocyte clone; epitope; stem cells; hematopoiesis; hematopoietic stem cell; major histocompatibility complex; cell selection; tetramer; paroxysmal nocturnal hemoglobinuria; hemoglobinuria, paroxysmal; cell surface; major histocompatibility antigen; cd1d; clonal selection; glycosylphosphatidylinositol; humans; human; priority journal; paroxysmal nocturnal haemoglobinuria (pnh); autoreactive t cells; glycosylphosphatidylinositol (gpi)
Journal Title: Leukemia
Volume: 15
Issue: 8
ISSN: 0887-6924
Publisher: Nature Publishing Group  
Date Published: 2001-08-01
Start Page: 1148
End Page: 1152
Language: English
DOI: 10.1038/sj.leu.2402180
PUBMED: 11480554
PROVIDER: scopus
DOI/URL:

http://www.scopus.com/inward/record.url?eid=2-s2.0-0034882347&partnerID=40&md5=dec7957bb4e408f35e9bf4cd4ebb034b
Notes: Export Date: 21 May 2015 -- Source: Scopus
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MSK Authors
  1. Anastasios Karadimitris
    13 Karadimitris
  2. Lucio Luzzatto
    105 Luzzatto
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