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Stem Cell Collection and Gene Transfer in Fanconi Anemia

13 Dec 2006

Abstract excerpt

Fanconi anemia (FA) is a rare genetic syndrome characterized by progressive bone marrow failure (BMF), congenital anomalies, and a predisposition to malignancy. Successful gene transfer into hematopoietic stem cells (HSCs) could reverse BMF in this disease. We developed clinical trials to determine whether a sufficient number of CD34(+) stem cells could be collected for gene modification and to evaluate the...

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PMID: 17164793
DOI: 10.1038/sj.mt.6300033

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