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Article

Rapid lentiviral transduction preserves the engraftment potential of Fanca(-/-) hematopoietic stem cells.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 Jun 2008

Müller Lars U W, Milsom Michael D, Kim Mi-Ok, Schambach Axel, Schuesler Todd, Williams David A

Abstract excerpt

Fanconi anemia (FA) is a rare recessive syndrome, characterized by congenital anomalies, bone marrow failure, and predisposition to cancer. Two earlier clinical trials utilizing gamma-retroviral vectors for the transduction of autologous FA hematopoietic stem cells (HSCs) required extensive in vitro manipulation and failed to achieve detectable long-term engraftment of transduced HSCs. As a strategy for...

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Identifiers

PMID: 18398427
DOI: 10.1038/mt.2008.67

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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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