RNAi Therapies as Possible Cure for TTR Amyloidosis and Other Diseases

TTR amyloidosis is a rare hereditary disease characterized by abnormal production of a protein called transthyretin, or TTR, which is responsible for shuttling the thyroid hormone thyroxine (T4) and retinol (an animal form of vitamin A) around the body. Hence, the name transthyretin: transports thyroxine and retinol. The defective version of the protein is unable to perform …