Gene therapy: Lessons learned from liver transplantation for transthyretin‐amyloidosis
Ole B. Suhr, Gösta Holmgren, Erik Lundgren – 19 November 2004 – Familial amyloidotic polyneuropathy is the common form of hereditary generalized amyloidosis and is characterized by the accumulation of amyloid fibrils in the peripheral nerves and other organs. Liver transplantation (LTx) has been utilized as a therapy for familial amyloidotic polyneuropathy because the variant transthyretin (TTR) is predominantly synthesized by the liver, but this therapy has several problems. Thus, we need to develop a new treatment that prevents the production of the variant TTR in the liver.