Prospects for gene therapy using haemopoietic stem cells

Best Practice & Research. Clinical Haematology
Leslie J Fairbairn, J C Ewing

Abstract

Gene therapy has thus far promised much and delivered little. Much of this has been due to deficiencies in the reagents and methodologies employed in early clinical trials. Recent technological advances in vectors and haemopoietic stem cell manipulation, coupled with improved pre-clinical assays of gene transfer and expression in re-populating stem cells give cause for greater optimism. Here we review these advances and indicate areas requiring further development before clinical gene therapy in the haemopoietic system becomes a widely applicable treatment modality.

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Citations

Oct 8, 2003·Molecular Therapy : the Journal of the American Society of Gene Therapy·Yan Ru SuSergio Fazio

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