Sickle Cell Disease
Nature have produced a range of interesting articles about sickle cell, gene editing, new-born screening and more. Read them all here: Link
A condition that affects many people of African descent is finally meeting its therapeutic match.
Every parent or carer wants the best for their children, but sometimes biological miscues obstruct that universal hope. This is especially the case with sickle-cell disease, a disorder in which red blood cells take on an abnormal shape described as a crescent or sickle. This malformation restricts oxygen delivery to the body, leading to severe pain and organ damage. The disease is found mainly in people of African descent, although hotspots also exist in India and the Middle East.
Sickle-cell disease arises from a single genetic mutation. This makes it an attractive target for gene therapy: replacing or repairing the mutated gene could provide a cure. At least ten trials of gene therapy for sickle cell are under way. Early data are encouraging, although safety questions linger, and progress could be accelerated by increasing the participation of people of African descent in studies of the disease.