Scientists from King’s College London, UCL, and Moderna Inc. have utilised mRNA technology, akin to Covid-19 vaccines, to create an effective therapy for a rare genetic liver disease in mice. The study highlights mRNA’s potential for treating rare diseases, specifically argininosuccinic aciduria, a rare metabolic disorder.
The research shows that mRNA corrected the disease model, offering an alternative to traditional gene therapy with modified viruses. The team plans to test the therapy in humans and is exploring its application for other rare diseases. Dr. Paolo Martini from Moderna emphasises the collaborative potential of mRNA technology against rare diseases, providing hope for conditions like argininosuccinic aciduria.