Repair Sickle Cell Gene With Stem Cells, Johns Hopkins University Study

Researchers have used a patient’s stem cells to correct the genetic alteration that causes sickle cell disease. The painful, disabling inherited blood disorder affects mostly African-Americans. In a test tube, the corrected stem cells were coaxed into becoming immature red blood cells that then turned on a normal version of the gene. The research team cautions that the work, done only in the laboratory, is years away from clinical use in patients, but should provide tools for developing gene therapies for sickle cell disease, or SCD, and a variety of other blood disorders.

MORE ON THIS TOPIC