Transthyretin amyloidosis is caused by deposition of hepatocyte-derived transthyretin amyloid in peripheral nerves and heart. A paper recently published in the New England Journal of Medicine reports the safety and efficacy of a potent antitransthyretin small interfering RNA (RNAi) encapsulated in lipid nanoparticles and injected in patients with transthyretin amyloidosis. The RNAi resulted in sustained reduction of transthyretin levels. This study establishes a proof of concept for RNAi therapy targeting messenger RNA transcribed from a disease-causing gene.
Posted by Nicola Brunetti-Pierri, MD