Genetic Treatments for Sickle Cell

April 20, 2016

(Scientific American) – For decades physicians have known that a few children like Ceniya have unusual genetic mutations that counteract the effects of the sickle-cell flaw. Researchers would like to re-create their uncommon physiology in everyone with sickle-cell anemia. Though not technically a cure, the compensatory treatment would spare many of the 300,000 infants around the world who are born every year with sickle cell and who often do not live beyond childhood. It would also make life a lot easier for the more than 70,000 individuals living with the disease in the U.S., who, despite treatment that mitigates some of the most serious effects of the condition, often die in their 40s.