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By protecting the mRNA in a microdroplet of lipids, scientists were able to inject the mice intravenously with the therapy and target their liver cells. Messenger RNA is a molecule that contains instructions that direct the cells to make proteins.

However, these viral systems can cause severe adverse effects, such as reactions from the patient's own immune system, meaning that they can't be rolled out widely.Ĭonsequently, the team wanted to investigate the possibility of using mRNA technology as an alternative solution. Until recently, gene therapy employed modified viruses to bring the therapeutic gene to the disease cells. Most of these treatments use gene therapy to switch out the faulty gene and replace it with a normal functioning one, to alleviate the disease. However, fewer than 5% of these conditions have approved therapies. Rare diseases usually result from errors in the patient's DNA and affect around 300 million people worldwide. We believe it can now do the same for rare diseases.' Messenger RNA therapies are also currently being investigated in other rare inherited metabolic diseases - propionic and methylmalonic acidaemias - in global clinical trials sponsored by Moderna, including at Great Ormond Street Hospital for Children.Ĭo-lead Principal Investigator, Dr Julien Baruteau (UCL Great Ormond Street Institute of Child Health), said: 'Messenger RNA has revolutionised the field of vaccines during the COVID-19 pandemic.

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Over the coming years, the team aims to trial the therapy in people. The condition occurs in approximately one in 100,000 newborns. Patients affected by the disease are found to also experience an imbalance of glutathione regulation, which is important for liver detoxification.

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 of the disease.Īrgininosuccinic aciduria is an inherited metabolic disorder that affects how the body breaks down protein - potentially leading to high levels of ammonia in the blood.