The research, published in Science Translational Medicine, found that messenger RNA (mRNA) could be used to correct a rare liver genetic disease known as argininosuccinic aciduria in a mouse model ...

Last year, it also won a £1.5 million ($1.8 million) grant from Innovate UK to develop an exosome-based therapy to treat argininosuccinic aciduria (ASA) – a rare life-threatening metabolic ...