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

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 predisposit...

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Main Authors: Walsh, C E, Nienhuis, A W, Samulski, R J, Brown, M G, Miller, J L, Young, N S, Liu, J M
Formáid: Artigo
Teanga:Inglês
Foilsithe: 1994
Ábhair:
Research Article
Rochtain Ar Líne:https://ncbi.nlm.nih.gov/pmc/articles/PMC295276/
https://ncbi.nlm.nih.gov/pubmed/7929819
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