Activating Complex Regions of the Genome to Treat Rare Diseases
Researchers at Duke University have developed a potential new treatment for rare genetic diseases, such as Prader-Willi syndrome, by activating a master epigenetic switch using CRISPR, a technology that allows scientists to modify DNA in living organisms. This approach enables genes from one parent, which are normally silenced, to compensate for defective genes from the other parent. In the case of Prader-Willi syndrome, which causes physical, mental and behavioral issues, including constant hunger, the researchers successfully activated suppressed genes in lab-grown stem cells and neurons.
This research is supported by the National Institutes of Health (NIH) and the National Science Foundation (NSF).