Although diseases of blood cells constitute a relatively smallproportion of clinical disorders, many valuable studies of genetransfer have relied on hematopoietic stem cells or their progeny.What features of these cells explain their appeal in gene therapy?Perhaps most important is their hierarchical pattern of development(Figure 1). Fewer than 1 in 100,000 bone marrow cells are pluripotent,self-renewing hematopoietic stem cells, but these rare cellsyield progeny that multiply by many hundredfold and, in theprocess, follow a particular blood-cell lineage (monocytic,lymphocytic, granulocytic, megakaryocytic, or erythrocytic).These committed progenitors then multiply by a hundredfold . . . [Full Text of this Article]
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From the Division of Bone Marrow Transplantation and the Cell and Gene Therapy Program, St. Jude Children's Research Hospital, Memphis, Tenn.
Address reprint requests to Dr. Brenner at the Cell and Gene Therapy Program, St. Jude Children's Research Hospital, 322 N. Lauderdale, Memphis, TN 38105-2794.
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