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Science study holds implications for gene therapy and stem cell biology

ctivated by the random insertion of a disabled retrovirus appear to have exactly this property: They can increase the multiplication rate of stem cells. While this puts these cells at risk to cause cancer, the majority of mice that were investigated in this study showed no signs of such side effects.

"This is a step forward in our research because it helps us understand which genes are involved in regulating stem cell growth," Dr. Baum said. "This is frontier research. In other words, to date, scientists know little about stem cells, particularly how they grow and what factors cause them to compete with other stem cells or turn into more stem cells versus differentiating into different kinds of stem cells.

"The next step would be to increase the availability of genes that enhance stem cell fitness using this novel approach. Simultaneously, we need to study whether similar genes regulate the behavior of human stem cells, because so far our insights are limited to mouse models." Dr. Baum said.

Scientists, in general, experiment with related gene vectors to treat severe inborn disorders or cancer. There continues to be an increasing interest in gene therapy research with blood stem cells because these cells can be extracted and manipulated and then reinserted to repopulate the entire blood system, as in leukemia treatment.

The process has the potential to treat inherited blood disorders, as demonstrated in an increasing number of patients. The new study published in Science suggests that enhanced fitness of stem cells could be a welcome side effect of current gene-based therapies, but also a potential risk factor for leukemia later in life. "We still need to learn how many risks we can tolerate in gene therapy, just as in other forms of experimental medicine," Dr. Baum said.

In 2003, Dr. Baum established a research team in the molecular and gene therapy program in the Division of Experimental Hematology at Cincinnati Children's u
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Source:Cincinnati Children's Hospital Medical Center


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