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Science study holds implications for gene therapy and stem cell biology

A study in the May 20 issue of Science holds mixed blessings for scientists who follow research in gene therapy and stem cell biology. The study, conducted by scientists from Cincinnati Children's Hospital Medical Center, and Hannover Medical School and Hamburg University in Germany, reveals important evidence that tells us more about how blood forming stem cells regenerate. On the other hand, scientists offer caution in regards to the side effects associated with genetic engineering of stem cells.

The study, whose lead author is Christopher Baum, MD, an adjunct associate professor of experimental hematology at Cincinnati Children's, is one of a family of studies on using disabled retroviruses as gene transfer vectors. To date, disabled retroviruses have mostly been tested experimentally in laboratory settings but are also being tested in well-controlled clinical trials to treat severe inborn diseases or cancer in humans.

Retrovirus-based gene transfer vectors are partially defective retroviruses that integrate into the genome of a cell, thus potentially increasing the activity of neighboring genes.

A previous study by Dr. Baum and team revealed that the insertion of several such retrovirus vectors into a single blood-forming stem cell can lead to leukemia in mice, because several "signaling" genes were activated that worked in concert to cause disease.

The question left: What happens if only one of these genes is activated in a stem cell?

This question is addressed in the newly published Science study. Researchers found that they could use the random genome insertion of disabled retroviruses as a tool to discover genes that enhance the fitness of stem cells. Such genes are of great interest for regenerative medicine because they can improve the prospects of stem cell-based therapies. Stem cells are very rare in the body and typically need to be multiplied for medical use. The genes that the researchers found to be marked and a
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Source:Cincinnati Children's Hospital Medical Center


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