Salk scientists modify CRISPR to epigenetically treat diabetes, kidney disease, muscular dystrophy

A research team led by Juan Carlos Izpisua Belmonte, and including co–first authors Hsin-Kai (Ken) Liao and Fumiyuki Hatanaka, developed a new version of the CRISPR/Cas9 gene-editing tool, allowing them to activate genes without creating DNA breaks. This breakthrough could circumvent a major hurdle to using gene editing in the treatment of human diseases. Most CRISPR/Cas9 systems create double-stranded DNA breaks, but many researchers are opposed to creating these breaks in humans because they can cause additional health problems. However, the system developed in the Belmonte lab alters gene expression without actually breaking DNA. The team used this new approach to treat several diseases in mouse models, including diabetes, acute kidney disease and muscular dystrophy. They are now working to improve the precision of their system and apply it to more cell types in the hopes of treating a wider range of human diseases, rejuvenating specific organs and reversing age-related conditions, such as hearing loss and macular degeneration.