Protection of stem cell-derived lymphocytes in a primate AIDS gene therapy model after in vivo selection.

Publication Type:

Journal Article


PloS one, Volume 4, Issue 11, p.e7693 (2009)


2009, Acquired Immunodeficiency Syndrome, AIDS Vaccines, Animals, Cell Line, Clinical Research Division, Gene Therapy, Hela Cells, Hematopoietic Stem Cells, HIV Infections, Humans, LYMPHOCYTES, Lymphoma, Macaca nemestrina, Mutation, Plasmids, Transgenes


There is currently no effective AIDS vaccine, emphasizing the importance of developing alternative therapies. Recently, a patient was successfully transplanted with allogeneic, naturally resistant CCR5-negative (CCR5Delta32) cells, setting the stage for transplantation of naturally resistant, or genetically modified stem cells as a viable therapy for AIDS. Hematopoietic stem cell (HSC) gene therapy using vectors that express various anti-HIV transgenes has also been attempted in clinical trials, but inefficient gene transfer in these studies has severely limited the potential of this approach. Here we evaluated HSC gene transfer of an anti-HIV vector in the pigtailed macaque (Macaca nemestrina) model, which closely models human transplantation.