Scientists Find Potential New Use for Cancer Drug in Gene Therapy for Blood Disorders

June 30, 2014

(Science Codex) – Scientists working to make gene therapy a reality have solved a major hurdle: how to bypass a blood stem cell’s natural defenses and efficiently insert disease-fighting genes into the cell’s genome. In a new study led by Associate Professor Bruce Torbett at The Scripps Research Institute (TSRI), a team of researchers report that the drug rapamycin, which is commonly used to slow cancer growth and prevent organ rejection, enables delivery of a therapeutic dose of genes to blood stem cells while preserving stem cell function.