Beam unveils new CRISPR basic machining tool for targeting sickle cell

RAt the moment, trillions of donut-shaped red blood cells are whispering through your veins and acid oxygen from your lungs to the tips of your fingers and toes and everywhere in between. But if you are one of the millions of people around the world who have a sickle cell disease, this constant commute is constantly interrupted.

The moment the hemoglobin molecules in the red blood cells of a sickle cell patient release their oxygen load, they begin to hold on to each other and get stuck in long, tortuous braids that stretch and twist the cells, deforming them into the eponymous “sickle” shape. When the red blood cells move through the lungs again and pick up a new oxygen charge, their hemoglobin molecules click again in solitary suspension. This can happen up to four times per minute. And this repeated cycle makes the red blood cells hard and fragile, causing them to break apart and block blood vessels, leading to painful crises, and if left untreated, death. A normal red blood cell can survive up to four months in circulation. For patients with sickle cell disease, their cells may not last a week.