Paroxysmal nocturnal haemoglobinuria: Nature's gene therapy?
Open Access
- 1 June 2002
- journal article
- review article
- Published by BMJ in Molecular Pathology
- Vol. 55 (3) , 145-152
- https://doi.org/10.1136/mp.55.3.145
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.Keywords
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