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Article

Phenotype correction of Fanconi anemia group A hematopoietic stem cells using lentiviral vector.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 Oct 2003

Yamada Kaoru, Ramezani Ali, Hawley Robert G, Ebell Wolfram, Arwert Fre, Arnold Larry W, Walsh Christopher E

Abstract excerpt

Fanconi anemia (FA) is an autosomal recessive disease characterized by progressive bone marrow failure due to defective stem cell function. FA patients' cells are hypersensitive to DNA cross-linking agents such as mitomycin C (MMC), exposure to which results in cytogenetic aberrations and cell death. To date Moloney murine leukemia virus vectors have been used in clinical gene therapy. Recently, third-generation...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 14529833
DOI: 10.1016/s1525-0016(03)00223-5

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026. Manifest publication-graph-cohort-2026-09-06.1.

Source receipt: pubmed, retrieved 6 Sept 2026.

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