Regular infusions of messenger RNA (mRNA) coding for a missing enzyme have shown to reduce life-threatening medical emergencies in individuals with propionic acidemia, a rare metabolic disease. This study by Moderna provides the first published clinical data demonstrating the potential of mRNA technology as a drug to replace missing proteins.
The innovative technology behind some of the most successful COVID-19 vaccines is showing signs of success in treating a rare but dangerous metabolic disease . Researchers report today in that regular infusions of messenger RNA (mRNA) coding for a missing enzyme may have slashed the rate of life-threatening medical emergencies in a handful of people with propionic acidemia , a condition in which toxic compounds build up in the body and damage organs.
The results from Moderna are the first published clinical data showing that mRNA, used in vaccines to deliver viral proteins that provoke an immune response, could potentially work as a drug to replace a protein people lack
Mrna Metabolic Disease Propionic Acidemia Drug Candidate Missing Enzyme Moderna
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