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
AUTHOR CONTACT:
Stanton L. Gerson
Case Western Reserve University, Cleveland, Ohio, USA.
Phone: 216-368-1177
Fax: 216-368-1166
E-mail: slg5@po.cwru.edu
View the PDF of this article at: http://www.jci.org/cgi/content/full/112/10/1561
RELATED ARTICLE:
TITLE: Methylguanine methyltransferase–mediated in vivo selection and chemoprotection of allogeneic stem cells in a large-animal model
AUTHOR CONTACT:
Hans-Peter Kiem
Fred Hutchinson Cancer Research Center, Seattle, Washington, USA.
Phone: 206-667-4425
Fax: 206-667-6124
E-mail: hkiem@fhcrc.org
View the PDF of this article at: http://www.jci.org/cgi/content/full/112/10/1581
ACCOMPANYING COMMENTARY:
Hematopoietic stem cell gene therapy: selecting only the best
AUTHOR CONTACT:
Arthur Bank
Columbia University, New York, New York, USA.
Phone: 212-305-4186
Fax: 212-923-2090
E-mail: ab13@columbia.edu
Journal of Clinical Investigation