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 ce lls engraft and proliferate extensively in the recipient. Arising bloo d cell progeny have the potential to provide the host with therapeutic levels of a desired gene product for at least two decades, quite poss ibly a lifetime. A long-standing challenge in human blood cell gene th erapy has been progression from adoptive transfer of allogeneic stem c ells which are inherently genetically ''correct'', to adoptive transfe r of autologous stem cells that have been specifically genetically ''c orrected''. 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 an d discussed.