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Researchers Find Potential New Use For Cancer Drug In Gene Therapy For Blood Disorders

Update Date: Jun 27, 2014 10:26 AM EDT
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Scientists working to make gene therapy a reality have achieved yet another milestone. They have figured out how to bypass a blood stem cell's natural defenses while efficiently inserting disease-fighting genes into the cell's genome. 

In a new study researchers reported that the drug rapamycin, which is commonly used to slow cancer growth and prevent organ rejection, enabled delivery of a therapeutic dose of genes to blood stem cells while preserving stem cell function. 

For a disease such as leukemia or leukodystrophy, where mutations in the DNA cause abnormal cell function, efficiently targeting the stem cells that produce these blood cells could be a successful approach to halting the disease and prompting the body to produce healthy blood cells, the press release added. 

"If you produce a genetic modification in your blood stem cells when you are five years old, these changes are lifelong," said lead researcher of the study Bruce Torbett at The Scripps Research Institute (TSRI), in the press release.

Torbett added that the gene-modified stem cells can develop into many types of cells that travel throughout the body to therapeutic effects. 

"We wanted to make sure the conditions we will use preserve stem cells, so if we transplant them back into our animal models, they act just like the original stem cells," said Torbett. "We showed that in two sets of animal models, stem cells remain and produce gene-modified cells."

Findings of the study has been published in the journal Blood. 

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