Scientists are seeing promising early results from the first studies testing gene editing for painful, inherited blood disorders that plague millions worldwide.
Victoria Gray, the first patient in the sickle cell study, had long suffered severe pain bouts that often sent her to the hospital. Both diseases involve mutations in a gene for haemoglobin, the substance in red blood cells that carries oxygen throughout the body. “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 haemoglobin again, said one study leader, Dr. Haydar Frangoul of the Sarah Cannon Research Institute in Nashville.
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