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Covid-19 vaccine technology to treat rare disease

Updated: Jun 17

Mice with liver cancer

A therapy, based on the same messenger RNA (mRNA) technology used in Covid-19 vaccines, has successfully corrected a rare liver disease in mice, and could potentially treat other types of rare disease.

Researchers at University College London (UCL), and King’s College London, both UK, and the pharmaceutical company Moderna, tested the approach on mice with argininosuccinic aciduria – a genetic condition affecting 1 in 100,000 newborns that impairs the body’s ability to break down proteins.

Because mRNA contains the ‘instructions’ for cells to make proteins, the team saw that injecting mRNA molecules into the liver of both newborn and older mice, with the disease, corrected some of its lethal effects, such as high ammonia levels in the blood.

In addition, the study in Science Translational Medicine, reported that while all untreated mice died within the first two weeks of life, treated mice survived for over three months.

Julien Baruteau, at UCL Great Ormond Street Institute of Child Health, said: “Messenger RNA has revolutionised the field of vaccines during the COVID-19 pandemic. We believe it can now do the same for rare diseases.”

Last week marked Rare Disease Day – it is estimated that these diseases affect about 300 million people worldwide 


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