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Article

Efficient retrovirus-mediated PIG-A gene transfer and stable restoration of GPI-anchored protein expression in cells with the PNH phenotype.

Blood - 15 May 2001

Nishimura Ji, Phillips K L, Ware R E, Hall S, Wilson L, Gentry T L, Howard T A, Murakami Y, Shibano M, Machii T, Gilboa E, Kanakura Y, Takeda J, Kinoshita T, Rosse W F, Smith C A

Abstract excerpt

Paroxysmal nocturnal hemoglobinuria (PNH) is a clonal hematopoietic stem cell disorder characterized by complement-mediated hemolysis due to deficiencies of glycosylphosphatidylinositol-anchored proteins (GPI-APs) in subpopulations of blood cells. Acquired mutations in the X-linked phosphatidylinositol glycan-class A (PIG-A) gene appear to be the characteristic and pathogenetic cause of PNH. To develop a gene...

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Source

Identifiers

PMID: 11342424
DOI: 10.1182/blood.v97.10.3004

Genes

GPI
NGFR

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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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