Fanconi Anemia

Cyclophosphamide, Irradiation May Enhance Gene Therapy

Published in Blood Weekly, March 22nd, 2001

by N.R. Saltmarsh, staff medical writer -- Nontoxic doses of therapies already used for Fanconi anemia may boost the effectiveness of gene therapy for the disease.

"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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Source: Blood Weekly (2001-03-22)

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