Sickle Cell Disease Corrected in Adult Mice

Researchers off hope for cure of sickle cell disease.

ScienceDaily — National Institutes of Health-funded scientists have corrected sickle cell disease in adult laboratory mice by activating production of a special blood component normally produced before, but not after, birth.

"This discovery provides an important new target for future therapies in people with sickle cell disease," said Susan B. Shurin, M.D., acting director of the NIH's National Heart, Lung, and Blood Institute, which co-funded the study. "More work is needed before it will be possible to test such therapies in people, but this study demonstrates that the approach works in principle."

Researchers at Harvard Medical School in Boston and the University of Texas at Austin corrected sickle cell disease in mice that had been bred to have the inherited blood disorder. The National Heart, Lung, and Blood Institute, the National Cancer Institute, and the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) -- all part of the NIH -- funded the research. The results of the study will appear online Oct. 13 in the journal Science.

Sickle cell disease results from an abnormality in hemoglobin, the protein found in red blood cells that is responsible for transporting oxygen throughout the body. People living with sickle cell disease have two copies of an altered gene that produces sickle hemoglobin instead of normal adult hemoglobin. Sickle hemoglobin changes shape after releasing its oxygen, causing the red blood cell to become stiff, misshapen and sticky, and slowing blood flow to tissues. This process damages organs and causes pain.