CRISPR editing aims to cut out the switching gene.“What we are doing is turning that switch back off and making the cells think they are back in utero, basically,” so they make fetal hemoglobin again, said one study leader, Dr.
Haydar Frangoul of the Sarah Cannon Research Institute in Nashville.The treatment involves removing stem cells from the patient's blood, then using CRISPR in a lab to knock out the switching gene.
Patients are given strong medicines to kill off their other, flawed blood-producing cells. Then they are given back their own lab-altered stem cells.
Saturday’s results were on the first 10 patients, seven with beta thalassemia and three with sickle cell. The two studies in Europe and the United States are ongoing and will.