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Article

Paroxysmal nocturnal haemoglobinuria: nature's gene therapy?

Molecular pathology : MP - 1 Jun 2002

Johnson R J, Hillmen P

Abstract excerpt

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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Share to Glycosylphosphatidylinositols
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Source

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Identifiers

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

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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