Stem cell collection and gene transfer in Fanconi anemia.

  title={Stem cell collection and gene transfer in Fanconi anemia.},
  author={Patrick F. Kelly and Susan Radtke and Christof von Kalle and Brenden Balcik and Kimberley Bohn and Robin Mueller and Todd Schuesler and Moira Haren and Lilith Reeves and Jose A. Cancelas and Thomas Leemhuis and Richard Harris and Arleen D. Auerbach and Franklin O. Smith and Stella M. Davies and David A Williams},
  journal={Molecular therapy : the journal of the American Society of Gene Therapy},
  volume={15 1},
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 safety and efficacy of HSC-corrective gene transfer in FA genotype A (FANCA) patients… CONTINUE READING
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