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Certain small RNAs silence the production of a particular protein — a phenomenon called RNA interference. In the amyloidoses, different proteins are overproduced, circulate in the blood, get deposited in various organs, and cause organ dysfunction. One protein that commonly causes hereditary amyloidosis, and some nonhereditary cases, is transthyretin — a carrier molecule for both thyroxine and vitamin A. Transthyretin is produced predominantly by hepatocytes. In its normal form, it deposits in tissues and might be an important cause of heart failure from senile systemic amyloidosis. In mutant forms, the protein deposits much more heavily in target organs.
An industry-sponsored, multinational team discovered a naturally occurring small interf…