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Article

Phenotypic correction of Fanconi anemia in human hematopoietic cells with a recombinant adeno-associated virus vector.

The Journal of clinical investigation - 1 Oct 1994

Walsh C E, Nienhuis A W, Samulski R J, Brown M G, Miller J L, Young N S, Liu J M

Abstract excerpt

Fanconi anemia (FA) is a recessive inherited disease characterized by defective DNA repair. FA cells are hypersensitive to DNA cross-linking agents that cause chromosomal instability and cell death. FA is manifested clinically by progressive pancytopenia, variable physical anomalies, and predisposition to malignancy. Four complementation groups have been identified, termed A, B, C, and D. The gene for the FA...

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Identifiers

PMID: 7929819
DOI: 10.1172/JCI117481
PMCID: PMC295276

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