Stem Cell-Based Gene Therapy

HSCs from different sources have different transduction efficiencies

Published in Gene Therapy Weekly, May 1st, 2003

Results of studies in rhesus macaques have led hematologists with the U.S. National Heart, Lung, and Blood Institute to recommend that "difference in transduction efficiency of HSCs collected from different sources should be considered whenever planning clinical gene therapy trials and should preferably be tested directly in comparative studies."

"Gene transfer experiments in nonhuman primates have been shown to be predictive of success in human clinical gene therapy trials. In most nonhuman primate studies, hematopoietic stem cells (HSCs) collected from the peripheral blood or bone marrow after administration of granulocyte colony-stimulating factor (G-PSF) + stem cell...

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Source: Gene Therapy Weekly (2003-05-01)

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