U.S. poised to approve first gene-editing treatment in breakthrough for sickle cell patients

“We are more or less training the cells to express and to produce more of this fetal hemoglobin,” said Dr. Markus Mapara, director of blood and marrow transplantation at NewYork-Presbyterian/Columbia University Irving Medical Center, who treated patients in the exa-cel trials. Blood stem cells are extracted, edited, and then infused back into the patient’s blood stream. “We are more or less training the cells to express and to produce more of this fetal hemoglobin,” said Dr. Mapara. Read the full storyhere.