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Article

Stem cell transplantation for paroxysmal nocturnal hemoglobinuria

31 May 2010

Abstract excerpt

Paroxysmal nocturnal hemoglobinuria (PNH) is a rareblood disease that results from clonal expansion of amultipotent hematolopoietic stem cell harboring a PIG-A mutation.1 The PIG-A gene product is required for the biosynthesis of glycophosphatidylinositol anchors, a glycol-ipid moiety that tethers dozens of proteins to lipid bilayer of cell membranes. Consequently, the PNH stem cell and its progeny have a...

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PMID: 20513803
DOI: 10.3324/haematol.2010.023176

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