TY - JOUR T1 - Paroxysmal nocturnal haemoglobinuria: Nature's gene therapy? JF - Molecular Pathology JO - Mol Pathol SP - 145 LP - 152 DO - 10.1136/mp.55.3.145 VL - 55 IS - 3 AU - R J Johnson AU - P Hillmen Y1 - 2002/06/01 UR - http://mp.bmj.com/content/55/3/145.abstract N2 - 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. ER -