Protection and selection for gene therapy in the hematopoietic system.
AffiliationCancer Research UK Gene Therapy Group, Paterson Institute for Cancer Research, Christie Hospital NHS Trust, Wilmslow Road, Manchester M20 4BX, UK.
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AbstractHematopoietic 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.
CitationProtection and selection for gene therapy in the hematopoietic system. 2004, 6 (2):133-46 J Gene Med
JournalThe Journal of Gene Medicine