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Review
. 2004 Feb;6(2):133-46.
doi: 10.1002/jgm.533.

Protection and selection for gene therapy in the hematopoietic system

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Review

Protection and selection for gene therapy in the hematopoietic system

Michael D Milsom et al. J Gene Med. 2004 Feb.

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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