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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הוצא לאור ב:	J Clin Invest
Principais autores:	Walsh, C E, Nienhuis, A W, Samulski, R J, Brown, M G, Miller, J L, Young, N S, Liu, J M
פורמט:	Artigo
שפה:	Inglês
יצא לאור:	American Society for Clinical Investigation 1994
נושאים:	Research Article
גישה מקוונת:	https://ncbi.nlm.nih.govhttps://pmc.ncbi.nlm.nih.gov/articles/PMC295276/ https://ncbi.nlm.nih.govhttps://pubmed.ncbi.nlm.nih.gov/7929819/ https://ncbi.nlm.nih.govhttps://doi.org/10.1172/JCI117481
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