Youthful cure for sickle cell disease

摘要

SWITCHING off a single gene could help treat sickle cell disease by keeping the blood forever young. The condition is caused by a mutant form of adult haemoglpbin, but not by fetal haemoglobin. Stuart Orkin of Harvard Medical School in Boston, and colleagues, effectively cured mice that normally develop a sickle cell-like condition by knocking out BCLiiA, the gene that controls the switchover from fetal to adult haemoglobin production (Science, DOI: 10.1126/ science.1211053).