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Article

Gene therapy of Fanconi anemia: preclinical efficacy using lentiviral vectors.

Blood - 15 Oct 2002

Galimi Francesco, Noll Meenakshi, Kanazawa Yoshiyuki, Lax Timothy, Chen Cindy, Grompe Markus, Verma Inder M

Abstract excerpt

Fanconi anemia (FA) is an inherited cancer susceptibility syndrome caused by mutations in a DNA repair pathway including at least 6 genes (FANCA, FANCC, FANCD2, FANCE, FANCF, and FANCG). The clinical course of the disease is dominated by progressive, life-threatening bone marrow failure and high incidence of acute myelogenous leukemia and solid tumors. Allogeneic bone marrow transplantation (BMT) is a therapeutic...

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Source

Identifiers

PMID: 12351379
DOI: 10.1182/blood-2002-04-1245

Genes

FANCA
FANCC
FANCD2
FANCE
FANCF
FANCG

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

Related research

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