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
Source:Cincinnati Children's Hospital Medical Center