UC Davis stem researchers demonstrate safety of gene therapy using adult ...(he significantly large number of study a...)

Date:5/6/2008

he significantly large number of study animals, our investigation further illuminates the safety of gene therapy using hematopoietic stem cells. It allows us to rest easier with the knowledge that if we insert corrective genes into human bone marrow stem cells and administer those cells to human patients, we are providing a relatively safe therapy."

Today, gene therapy using the adult blood-forming cells found in bone marrow (known as hematopoietic stem cells) is normally done in an autologous transplant setting. The therapy involves taking hematopoietic stem cells from a person who needs treatment, genetically modifying the cells perhaps by adding a missing gene and then re-inserting the cells back into the same person. This treatment eliminates the complications of graft-versus-host disease or host rejection that can occur in allogeneic transplants (therapy where the cell donor is different from the recipient).

In the current study, 630 immunodeficient mice received mesenchymal stem cells from one human bone marrow donor and hematopoietic stem cells from another. Mesenchymal stem cells are a type of cell found in bone marrow that support the function of hematopoietic stem cells, and can give rise to bone, cartilage, fat, and muscle. Genes were inserted into the hematopoietic stem cells using one of two viral vectors either a retrovirus or a lentivirus before they were transplanted into the mice, along with genetically modified mesenchymal stem cells.

Of the mice used in the long-term study, four developed human leukemia. None of those mice, however, were found to have vector DNA present in the malignant cells.

"This is evidence of a natural tendency for human bone marrow stem cells to develop leukemia in long-term studies, not that the leukemia was caused by the genetic modification," Bauer explained. "It's a good statistical control for our method."

Bauer noted that while the current study results are important f
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Contact: Charles Casey charles.casey@ucdmc.ucdavis.edu 916-734-9048 University of California - Davis - Health System Source:Eurekalert

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