Adoptive transfer of genetically modified hematopoietic stem cells

Adoptive transfer of bone marrow derived blood forming stem cells is a well-recognized treatment option for some human genetic diseases. In this therapeutic approach a relatively small number of donor marrow cells engraft and proliferate extensively in the recipient. Arising blood cell progeny have the potential to provide the host with therapeutic levels of a desired gene product for at least two decades, quite possibly a lifetime. A long-standing challenge in human blood cell gene therapy has been progression from adoptive transfer of allogeneic stem cells which are inherently genetically 'correct', to adoptive transfer of autologous stem cells that have been specifically genetically 'corrected'. The progress in meeting this challenge will be reviewed. A promising approach for adoptive transfer of hematopoietic stem cells genetically modified in long-term marrow cultures will be presented and discussed.