Method of genetically modifying very primitive quiescent human hematopoietic stem cells

Patent Number: 6919209

Gene therapy is limited by ability to transfect quiescent cells. Since the very primitive hematopoietic stem cells reside in a quiescent state, it is difficult to transfect them. This patent demonstrates transfection of early hematopoietic stem cells using an adeno-associated vector. The patent has one independent claim that covers:

A method for stably transferring DNA into multi-potential hematopoietic stem cells in the G0 phase of the cell cycle, which comprises transducing said multi-potential hematopoietic stem cells with an adeno-associated virus vector that contains said DNA, wherein the transferred DNA remains integrated into the genome of the multi-potential hematopoietic stem cells for at least 4 weeks, wherein the multi-potential hematopoietic stem cells are maintained in the presence of cytokines IL-3, IL-6 and stem cell factor, and wherein the levels of said cytokines are about 10 ng/ml IL-3, about 10 ng/ml IL-6 and 1 ng/ml stem cell factor.

View this patent on the USPTO website.