Bone marrow: target for gene transfer

Marrow cells are currently the most widely utilized target in human trials of gene therapies, and hematopoietic stem cells may be the best target of all. Able both to self-replicate and to engender blood cells in all lineages, they could be a lifelong source of therapeutic genes for disorders in any lineage. Hematopoietic stem cells may also portend the therapeutic use of even more versatile embryonic stem cells.