Molecular approaches to the treatment of Fanconi anemia: recent advances.
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 consists 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 hypersensitivity 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.
Otsuki
T
T
Hematology Branch, NHLBI, National Institutes of Health, Bethesda, MD 20892, USA.
Liu
J M
JM
D002986:Clinical Trials as Topic; D005199:Fanconi Anemia / Q000235:genetics / Q000628:therapy*; D005808:Genes, Recessive; D015316:Genetic Therapy; D006801:Humans