Hereditary transthyretin (ATTRv) amyloidosis is a progressive, disabling, fatal disease caused by extracellular deposits in organs and tissues of misfolded transthyretin variants and wild-type transthyretin protein. The disease has various clinical manifestations, including cardiomyopathy and polyneuropathy. Drugs targeting hepatic messenger RNA (mRNA) can slow the progression of ATTRv polyneuropathy, because the liver produces 90% of the amyloidogenic protein. The specific interaction between the TTR silencer drugs and the TTR mRNA leads to the breakdown of mRNA, preventing the production of the transthyretin protein. TTR gene silencers have become a powerful therapeutic tool for ATTRv polyneuropathy.
Source: JAMA Online First