SickKids researchers ‘model’ future of precision medicine for rare inherited disorders

A team of researchers at The Hospital for Sick Children (SickKids) has successfully implemented a genome-editing technique in mice that could eventually be used in humans to treat almost 10 per cent of all rare inherited disorders. The team created a mouse model emulating the exact genetic mutation of a patient with Duchenne muscular dystrophy (DMD) and used a CRISPR-Cas9 strategy that restored full functionality to the disease-causing gene.