RT Journal Article
SR Electronic
T1 Paroxysmal nocturnal haemoglobinuria: Nature's gene therapy?
JF Molecular Pathology
JO Mol Pathol
FD BMJ Publishing Group Ltd and Association of Clinical Pathologists
SP 145
OP 152
DO 10.1136/mp.55.3.145
VO 55
IS 3
A1 R J Johnson
A1 P Hillmen
YR 2002
UL http://mp.bmj.com/content/55/3/145.abstract
AB 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.