Genetic treatments for sickle cell
May 01, 2016
Patients with sickle cell disease with high levels of fetal hemoglobin have a milder course of the
disease because the mutation that causes red blood cells to “sickle” resides
only in adult hemoglobin. Now researchers at Dana-Farber/Boston Children's Cancer and Blood Disorders Center and other centers are looking to gene editing or gene therapy
– and eventually a pill – to control the switch to ease the devastating
disease, Scientific American reports.