Protection and selection for gene therapy in the hematopoietic system |
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| Authors: | Milsom Michael D Fairbairn Leslie J |
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| Affiliation: | Cancer Research UK Gene Therapy Group, Paterson Institute for Cancer Research, Christie Hospital NHS Trust, Wilmslow Road, Manchester M20 4BX, UK. |
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| Abstract: | Hematopoietic stem cell gene therapy is potentially curative for a number of inherited and acquired disorders. However, poor gene transfer and expression in repopulating hematopoietic stem cells attenuate this potential. Here we review potential means of conferring a selective advantage to hematopoietic stem cells and their progeny, and discuss the issues that surround the use of selective advantages in vivo. |
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| Keywords: | gene therapy hematopoiesis selection drug resistance chemical inducers of dimerisation HoxB4 |
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