Effective Gene Therapy — In a paper recently published in Blood, Dr. Eric Bouhassira and his student Chanjung Chang describe a novel approach for the safe insertion of therapeutic genes into induced pluripotent stem cells (iPSCs). Using this new method, the duo was able to insert the α-globin gene into iPSCs to treat α-thalassemia, a blood-related disease that is usually fatal at birth and results from a mutation in the α-globin gene. After differentiation of the corrected-iPS into red blood cells, Dr. Bouhassira and Mr. Chang were able to demonstrate that the gene correction was very effective since red blood cells produced from the treated stem cells expressed quasi-normal levels of hemoglobins. These experiments represent a major step toward curing a-thalassemias and other blood diseases by transplantation of patient-specific, gene-corrected stem cells. Dr. Bouhassira is professor of medicine (hematology) and of cell biology, and the Ingeborg and Ira Leon Rennert Professor of Stem Cell Biology and Regenerative Medicine.
Posted on: Monday, October 15, 2012