Transthyretin amyloidosis is a progressive, life-threatening disease caused by the accumulation of misfolded transthyretin, predominantly in the nerves and heart. In this Review, Aimo and colleagues provide an update on the treatment strategies aimed at blocking transthyretin production, including small interfering RNAs, antisense oligonucleotides and gene editing approaches.
- Alberto Aimo
- Vincenzo Castiglione
- Michele Emdin
