Literature DB >> 12032224

Paroxysmal nocturnal haemoglobinuria: nature's gene therapy?

R J Johnson1, P Hillmen.   

Abstract

The development of paroxysmal nocturnal haemoglobinuria (PNH) requires two coincident factors: somatic mutation of the PIG-A gene in one or more haemopoietic stem cells and an abnormal, hypoplastic bone marrow environment. When both of these conditions are met, the fledgling PNH clone may flourish. This review will discuss the pathophysiology of this disease, which has recently been elucidated in some detail.

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Year:  2002        PMID： 12032224      PMCID： PMC1187166          DOI： 10.1136/mp.55.3.145

Source DB:  PubMed          Journal:  Mol Pathol        ISSN： 1366-8714


  63 in total

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Journal:  Blood       Date:  1992-03-15       Impact factor: 22.113

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Journal:  Lancet       Date:  1996-08-31       Impact factor: 79.321

3.  Variations in the red cells in paroxysmal nocturnal haemoglobinuria.

Authors:  W F Rosse
Journal:  Br J Haematol       Date:  1973-03       Impact factor: 6.998

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Journal:  Annu Rev Biochem       Date:  1993       Impact factor: 23.643

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Authors:  G Tremml; C Dominguez; V Rosti; Z Zhang; P P Pandolfi; P Keller; M Bessler
Journal:  Blood       Date:  1999-11-01       Impact factor: 22.113

6.  Complement-induced vesiculation and exposure of membrane prothrombinase sites in platelets of paroxysmal nocturnal hemoglobinuria.

Authors:  T Wiedmer; S E Hall; T L Ortel; W H Kane; W F Rosse; P J Sims
Journal:  Blood       Date:  1993-08-15       Impact factor: 22.113

7.  Circulating primitive stem cells in paroxysmal nocturnal hemoglobinuria (PNH) are predominantly normal in phenotype but granulocyte colony-stimulating factor treatment mobilizes mainly PNH stem cells.

Authors:  R J Johnson; A C Rawstron; S Richards; G J Morgan; D R Norfolk; S O Hillmen
Journal:  Blood       Date:  1998-06-15       Impact factor: 22.113

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Authors:  M G Low
Journal:  FASEB J       Date:  1989-03       Impact factor: 5.191

9.  Natural history of paroxysmal nocturnal hemoglobinuria.

Authors:  P Hillmen; S M Lewis; M Bessler; L Luzzatto; J V Dacie
Journal:  N Engl J Med       Date:  1995-11-09       Impact factor: 91.245

10.  Glycosylphosphatidylinositol-anchor-deficient mice: implications for clonal dominance of mutant cells in paroxysmal nocturnal hemoglobinuria.

Authors:  K Kawagoe; D Kitamura; M Okabe; I Taniuchi; M Ikawa; T Watanabe; T Kinoshita; J Takeda
Journal:  Blood       Date:  1996-05-01       Impact factor: 22.113
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  3 in total

1.  PIG-A mutations in normal hematopoiesis.

Authors:  Rong Hu; Galina L Mukhina; Steven Piantadosi; Jamie P Barber; Richard J Jones; Robert A Brodsky
Journal:  Blood       Date:  2005-02-01       Impact factor: 22.113

2.  Acute renal failure in a patient with severe hemolysis.

Authors:  Onur Kirkizlar; Mehmet Kendir; Zeynep Karaali; Umit Ure; Gulsen Ozbay; Dogan Selcuk; Rumeyza Kazancioglu
Journal:  Int Urol Nephrol       Date:  2007-01-19       Impact factor: 2.370

3.  Paroxysmal nocturnal hemoglobinuria in a girl with hemolysis and "hematuria".

Authors:  Zdenek Dolezel; Dana Dostalkova; Jan Blatny; Jiri Starha; Hana Gerykova
Journal:  Pediatr Nephrol       Date:  2004-07-20       Impact factor: 3.714
  3 in total

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