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(St. Jude Reference #)
|In Vivo Selection of Primitive Hematopoietic Cells
|Description||This invention describes a method that selects genetically modified hematopoietic progenitor or stem cells from unmodified hematopoietic cells. This is achieved by introducing a mutant dihydrofolate reductase (DHFR) gene that confers antifolate resistance into hematopoietic progenitor or stem cells and then administering an antifolate, which inhibits the growth of unmodified cells and a nucleoside transport inhibitor to block the salvage pathway. Cells that do not have the mutant DHFR gene cannot synthesize new nucleosides and the salvage for exogenous nucleosides is also blocked; therefore growth of unmodified cells is arrested. The invention also includes nucleic acids which encode mutant DHFR genes and hematopoietic cells transformed with such mutant genes.|
|Keywords||Gene therapy, DHFR|
|Granted Patents or Published Applications||US Patent No. 6,500,421|
|Related Scientific References||Allay, JA., et al., "In vivo Selection of Retrovirally Transduced Hematopoietic Stem Cells" Nature Medicine 4: 1136-1143 (1998)|
|Licensing Opportunities||We are seeking licensees in all fields for the development of this technology.|
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Last update: June 2006