New sickle cell disease gene therapies depend on getting the right mouse

Sickle cell disease is an extremely debilitating condition that affects up to 40% of the population in African countries, with patients suffering episodes of excruciating pain, organ damage and reduced life expectancy. This disease is caused by a mutation in a gene that makes hemoglobin, the protein that carries oxygen in red blood cells, with the damaged hemoglobin distorting the shape of red blood cells, causing painful and potentially life-threatening blockages in blood vessels. However, scientists have realized that increasing the production of a healthy form of this protein (fetal hemoglobin, which is usually only produced when we are in the womb), could provide a revolutionary treatment for these patients.