Simulation of hematopoiesis: implications for the gene therapy of lysosomal enzyme disorders.

doi:10.1159/000203880

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Simulation of hematopoiesis: implications for the gene therapy of lysosomal enzyme disorders.

Acta haematologica - 1 Jan 1996

Abkowitz J L, Persik M T, Catlin S N, Guttorp P

Abstract excerpt

Although the hematopoietic stem cell is an attractive target for gene transfer, little is known about its biology in vivo in large animals (including humans). We have studied the in vivo behavior of hematopoietic stem cells in glucose-6-phosphate dehydrogenase heterozygous (female Safari) cats, and demonstrated that clonal instability persists for up to 4.5 years after autologous marrow transplantation. This...

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