Fanconi Anemia
Study supports benefit of lentiviral-vectored gene therapy
Published in Gene Therapy Weekly, January 2nd, 2003
"Fanconi anemia (FA) is an inherited cancer susceptibility syndrome caused by mutations in a DNA repair pathway including at least six genes (FANCA, FANCC, FANCD2, FANCE, FANCF, and FANCG). The clinical course of the disease is dominated by progressive, life-threatening bone marrow failure and high incidence of acute myelogenous leukemia and solid tumors. Allogeneic bone marrow transplantation (BMT) is a therapeutic option but requires HLA-matched donors," researchers in the United States report.
"Gene therapy holds great promise for FA, but previous attempts to use retroviral vectors in humans have proven ineffective given the impaired proliferation potential of...
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Source: Gene Therapy Weekly (2003-01-02)
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