Highly efficient transfer and expression of MGMT into relatively few HSCs using a lentivirus vector led to repopulation of the hematopoietic compartment with gene-corrected cells following suitable drug treatment. This selection system may be useful in human clinical trials of gene therapy in bone marrow transplantation settings.
In an accompanying commentary, Arthur Bank from Columbia University College of Physicians and Surgeons discusses the potential of these results in significantly advancing our knowledge and ability to perform human HSC gene therapy.
TITLE: In vivo selection of MGMT(P140K) lentivirus-transduced human NOD/SCID repopulating cells without pretransplant irradiation conditioning
Stanton L. Gerson
Case Western Reserve University, Cleveland, Ohio, USA.
View the PDF of this article at: http://www.jci.org/cgi/content/full/112/10/1561
TITLE: Methylguanine methyltransferase-mediated in vivo selection and chemoprotection of allogeneic stem cells in a large-animal model
Fred Hutchinson Cancer Research Center, Seattle, Washington, USA.
View the PDF of this article at: http://www.jci.org/cgi/content/full/112/10/1581
Hematopoietic stem cell gene therapy: selecting only the best
Columbia University, New York, New York, USA.