New method reveals how mutations drive transthyretin amyloidosis and guides precision drug design

An international research team reveals new molecular mechanisms associated with pathogenic mutations in the protein transthyretin that cause transthyretin amyloidosis (ATTR), a group of fatal progressive diseases. The results, obtained thanks to a new methodological approach, open the door to the development of drugs with higher therapeutic potential, designed specifically for the variants of the protein associated with the disease.