Molecular Approaches to the Treatment of Fanconi Anemia : Recent Advances
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概要
- 論文の詳細を見る
Fanconi anemia (FA) is an autosomal recessive disorder that leads to aplastic anemia. Cells from FA patients are abnormally sensitive to DNA cross-linking agents such as mitomycin C. FA con-sists of at least five subgroups (FA-A through -E). The genes defective in the FA-C and FA-A groups have recently been cloned. Transfection of the normal FA gene into mutant cells corrects the hyper-sensitivity to DNA cross-linking agents and improves cell viability in vitro. The function of the FA gene products is still unclear, however. For patients lacking a compatible bone marrow transplantation donor, an experimental trial of gene therapy for group C FA is ongoing at the National Institutes of Health.
- The Keio Journal of Medicineの論文
- 1998-03-01
著者
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Otsuki Tetsuya
Hematology Branch Nhlbi National Institutes Of Health
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Otsuki Tetsuya
Hematology Branch National Heart Lung And Blood Institute National Institutes Of Health
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LIU Johnson
Hematology Branch, NHLBI, National Institutes of Health
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Liu Johnson
Hematology Branch Nhlbi National Institutes Of Health
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Liu Johnson
Hematology Branch National Heart Lung And Blood Institute National Institutes Of Health
関連論文
- Molecular Approaches to the Treatment of Fanconi Anemia : Recent Advances
- Assessment of mitomycin C sensitivity in Fanconi anemia complementation group C gene (Fac) knock-out mouse cells