mRNA Therapy Explained: The Future of Protein Replacement

mRNA therapy is an emerging treatment approach designed to help the body produce missing or dysfunctional proteins caused by genetic disorders. By delivering the blueprint cells need to make these proteins, researchers hope to reduce symptoms, improve cell function, and potentially change the course of certain diseases, including Cystic Fibrosis, Gaucher disease, and Familial Hypercholesterolemia. This video is brought to you by the National Organization for Rare Disorders (NORD).