Molecular Tweezers Targeting Transthyretin Amyloidosis

Transthyretin Amyloidosis – an Overview

Familial amyloidosis describes a group of late-onset autosomal dominant diseases with amyloid protein deposition occurring in various tissues throughout the body and in the bloodstream (reviewed in (1)). Transthyretin Amyloidosis (ATTR), the most common familial amyloidosis, is associated with mutations in the TTR gene and is generally characterized by progressive neuropathy, cardiomyopathy, ne...

A Guide to Transthyretin Amyloidosis

Wild Type Transthyretin Amyloidosis ATTRwt

Liver transplantation and transthyretin amyloidosis.

Liver transplantation as a specific treatment of transthyretin amyloidosis was first performed in 1990. The rationale for this treatment was that removal of the source (liver) of the amyloid precursor protein (mutated transthyretin) would stop progression of the disease. Indeed, after orthotopic liver transplantation (OLT), mutant transthyretin (TTR) is rapidly cleared from circulation. In the ...

Transthyretin amyloidosis and the kidney.

The amyloidoses are protein-misfolding disorders associated with progressive organ dysfunction. Immunoglobulin light chain is the most common, amyloid A the longest recognized, and transthyretin-associated amyloidosis (ATTR) the most frequent inherited systemic form. Although ATTR, an autosomal-dominant disease, is associated with at least 100 different transthyretin (TTR) mutations, the single...