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Article

Phenotypic correction of Fanconi anemia cells in the murine bone marrow after carrier cell mediated delivery of lentiviral vector.

Stem cell research & therapy - 19 Nov 2016

Chakkaramakkil Verghese Santhosh, Goloviznina Natalya A, Kurre Peter

Abstract excerpt

Fanconi anemia (FA) is an autosomal-recessive disorder associated with hematopoietic failure and it is a candidate for hematopoietic stem cell (HSC)-directed gene therapy. However, the characteristically reduced HSC numbers found in FA patients, their ineffective mobilization from the marrow, and re-oxygenation damage during ex vivo manipulation have precluded clinical success using conventional in vitro...

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Identifiers

PMID: 27865213
DOI: 10.1186/s13287-016-0431-z
PMCID: PMC5116221

Genes

FANCC

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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