Fanconi Anemia

Gene Therapy May Hold Promise for Phenotypic Correction

Published in Gene Therapy Weekly, March 16th, 2000

Gene transfer produces phenotypic correction of Fanconi anemia in group C knockout mice, researchers from the United States reported.

"Fanconi anemia (FA) is a genetic disorder characterized by bone marrow failure, congenital anomalies, and a predisposition to malignancy," wrote K.A. Gush and colleagues, University North Carolina, USA. "FA cells demonstrate hypersensitivity to DNA cross-linking agents, such as mitomycin C (MMC). Mice with a targeted disruption of the FANCC gene (fancc-/- nullizygous mice) exhibit many of the characteristic features of FA and provide a valuable tool for testing novel therapeutic strategies."

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Source: Gene Therapy Weekly (2000-03-16)

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