Article
Survival of the fittest: in vivo selection and stem cell gene therapy.
Blood - 1 Mar 2006
Neff Tobias, Beard Brian C, Kiem Hans-Peter
Abstract excerpt
Stem cell gene therapy has long been limited by low gene transfer efficiency to hematopoietic stem cells. Recent years have witnessed clinical success in select diseases such as X-linked severe combined immunodeficiency (SCID) and ADA deficiency. Arguably, the single most important factor responsible for the increased efficacy of these recent protocols is the fact that the genetic correction provided a selective...
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