In a breakthrough study of sickle cell disease, biomedical engineers have revealed that the building blocks of the disease are much less efficient at organizing than previously thought. The findings open the door to new treatments, including new medicines that could be prescribed at lower doses, for the approximately 20 million people worldwide who suffer from the lifelong disease.

The findings open the door to new treatments, including new medicines that could be prescribed at lower doses, for the approximately 20 million people worldwide who suffer from the lifelong disease.

The study, which includes the most precise measurements ever of the disease at the molecular level, is published in Science Advances, a journal of the American Association for the Advancement of Science.

«Even though it has been known for decades what causes sickle cell disease at the molecular level, no one has ever studied the disease at this level of detail,» said David Wood, an associate professor of biomedical engineering at the University of Minnesota and a lead author of the study. «What we found at the nanoscale was quite surprising. We found that the disease self-assembly process is less efficient than we thought, which means that it could be easier to develop new medicines that would be effective at lower doses and would cause fewer side effects for patients.»

Sickle cell disease is an inherited lifelong disorder that causes problems in the protein within red blood cells, called hemoglobin. The hemoglobin molecules carry oxygen throughout the body. With sickle cell disease, hemoglobin molecules form into fibers that act like stiff rods within the red blood cells. The formation of these fibers stiffens the red blood cells and can change the shape from disc-shaped to crescent, or sickle, shape.

When the red blood cells stiffen, they contribute to blockages in blood vessels that slow or stop the flow of blood. When this happens, oxygen can’t reach nearby tissues. The lack of oxygen can affect the entire body causing severe pain, increasing the risk of strokes, and causing infections.

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