Advances in Studying the Pathologic Mechanisms and Treatment Strategies of Transthyretin Amyloidosis.

Transthyretin amyloidosis (ATTR) is characterized by the deposition of unstable transthyretin protein (TTR) in the heart or peripheral nerves. Therapeutic strategies for ATTR include inhibition of the secretion of abnormal TTR by the liver, reducing the concentration of aberrant TTR in the circulation, and eliminating amyloid deposits of TTR in tissues. This article delves into the pathogenesis of TTR secretion from the liver into the bloodstream, its deposition in tissues, and the subsequent development of ATTR. In addition, we delineated the advancements in treatment strategies and discussed future research directions to provide novel insights for the identification of diagnostic and preventive targets.