A team of scientists from UCL, King’s College London, and Moderna has used technology from Covid-19 vaccines to create an effective therapy for a rare liver disease in mice. The therapy uses messenger RNA (mRNA) to correct a genetic disorder called argininosuccinic aciduria, that affects how the body breaks down protein. The researchers are planning to test the therapy on people with the aim of treating other rare inherited metabolic diseases. This mRNA therapy could be a safer alternative to traditional gene therapy that uses modified viruses. In the mouse model, the treatment successfully corrected the lethal consequences of the disease and enabled the mice to survive for longer. Researchers hope to apply this approach to other inherited liver diseases and translate it to human patients, especially children. The study was funded by various organizations including Moderna, the Medical Research Council, and the National Institute for Health and Care Research (NIHR) Great Ormond Street Hospital Biomedical Research Centre.


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