Mystery Solved: New Research Reveals Why a Key Protein Turns Deadly in Aging Bodies
By Scripps Research Institute
January 30, 2025
These insights could drive the development of new drugs to treat transthyretin amyloidosis, a progressive and fatal disease.
Transthyretin, a small but crucial protein, plays a vital role in transporting hormones through the blood and spinal fluid. However, when it misfolds after secretion, it can lead to serious health problems. Misfolded transthyretin forms toxic clumps in the heart and along nerves, causing transthyretin amyloidosis (ATTR)—a progressive and often fatal disease. ATTR affects up to 25% of men over 80, leading to symptoms such as shortness of breath, dizziness, and numbness or tingling in the extremities.
In a breakthrough study, researchers at Scripps Research have revealed new structural insights into transthyretin. Their findings, published in Nature Structural & Molecular Biology, show how the protein’s three-dimensional asymmetry may contribute to its instability. This discovery could pave the way for new drug treatments to combat ATTR.