Stem Cells Used to Target Defective Genes

by Brian Lee

Genetic defects may be repaired through a technique created by researchers from the Morgridge Institute for Research and Northwestern University, the organizations announced this week.

In an article published in the Proceedings of the National Academy of Sciences, the team described using human pluripotent stem cells and DNA-cutting protein from meningitis bacteria to target and repair defective genes.

“With this system, there is the potential to repair any genetic defect, including those responsible for some forms of breast cancer, Parkinson’s and other diseases,” Zhonggang Hou of the Morgridge Institute said in a statement. “The fact that it can be applied to human pluripotent stem cells opens the door for meaningful therapeutic applications.”

Other team members included James Thomson of the Morgridge Institute, and Erik Sontheimer and Yan Zhang of Northwestern.