Endocrine Abstracts

Transthyretin (TTR) is a plasma and cerebrospinal fluid (CSF)-circulating protein. Besides the primordially attributed systemic role as transporter molecule of thyroxine (T₄) and retinol (through the binding to retinol-binding protein (RBP)), TTR has been recognised as a protein with important functions in several aspects of the nervous system physiology. TTR has been shown to play an important role in behaviour, cognition, amidated neuropeptide processing, and nerve regeneration. Further, it has been proposed that TTR is neuroprotective in Alzheimer’s disease and cerebral ischemia. Mutations in TTR are a well-known cause of familial amyloidotic polyneuropathy (FAP), an autosomal dominant neurodegenerative disorder characterised by systemic deposition of TTR amyloid fibrils, particularly in the peripheral nervous system. The purpose of this review is to highlight the roles of TTR in the nervous system, beyond its systemic role as transporter molecule of T₄ and RBP-retinol.