Published in Blood Weekly, March 22nd, 2001
"Fanconi anemia (FA) is an autosomal recessive disorder characterized by birth defects, increased incidence of malignancy, progressive bone marrow failure, and cellular hypersensitivity to DNA cross-linking agents," explained M. Noll and colleagues writing in Molecular Therapy.
FA patients who are candidates for bone marrow transplantation may also benefit from hematopoietic gene therapy, but a treatment used in mice with a FA-like syndrome was considered too risky to try in...
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