Cancer Drug Rapamycin May Lead To Gene Therapy For Blood Disorders
Friday, June 27, 2014 - 19:50
in Health & Medicine
Scientists working to make gene therapy a reality say they have figured out how to bypass a blood stem cell's natural defenses and efficiently insert disease-fighting genes into the cell's genome. 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. The findings in Blood could lead to more effective and affordable long-term treatments for blood cell disorders in which mutations in the DNA cause abnormal cell functions, such as in leukemia and sickle cell anemia. read more