Prospects for gene therapy using haemopoietic stem cells

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...

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Published inBest practice & research. Clinical haematology Vol. 14; no. 4; pp. 823 - 834
Main Authors Fairbairn, Leslie J, Ewing, Joanne C
Format Journal Article
LanguageEnglish
Published Netherlands Elsevier Ltd 01.12.2001
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Summary: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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ISSN:1521-6926
1532-1924
DOI:10.1053/beha.2001.0175