Article
In vivo selection of wild-type hematopoietic stem cells in a murine model of Fanconi anemia.
Blood - 15 Sept 1999
Battaile K P, Bateman R L, Mortimer D, Mulcahy J, Rathbun R K, Bagby G, Fleming W H, Grompe M
Abstract excerpt
Fanconi anemia (FA) is an autosomal recessive disorder characterized by birth defects, increased incidence of malignancy, and progressive bone marrow failure. Bone marrow transplantation is therapeutic and, therefore, FA is a candidate disease for hematopoietic gene therapy. The frequent finding of somatic mosaicism in blood of FA patients has raised the question of whether wild-type bone marrow may have a...
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