Gene Editing Tool Holds Promise in Rare Disease Treatments

The gene editing technology known as CRISPR has won recognition as a powerful research tool, but a new study from scientists at The Hospital for Sick Children in Toronto is hailing the technology for its potential therapeutic applications. The study, published in the December 10 online edition of the American Journal of Human Genetics, shows how researchers, for the first time, used CRISPR to remove a duplicated gene from a genome and restored the function in the gene that causes Duchenne muscular dystrophy. We spoke to principal investigator of the study Ronald Cohn, Chief of Clinical and Metabolic Genetics and Co-director of the Centre for Genetic Medicine at SickKids, about the study, the next steps to advance the work, and the potential of the gene editing technology to be used in the treatment of a broad range of genetic diseases.