Diagram of messenger ribonucleic acid (mRNA)
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Biotech company Moderna announced May 19 that 16 people have benefited from an experimental mRNA therapy that replaces the defective enzyme behind a rare genetic disease. While this is good news for patients with this disease, its significance extends further. If this kind of mRNA therapy proves safe and effective, it could help treat a wide range of diseases and even revolutionize medicine.
People over the age of 1 in the trial have a disease called propionic acid.
