Structure-based design of kinetic stabilizers that ameliorate the transthyretin amyloidoses

S Connelly, S Choi, SM Johnson, JW Kelly… - Current opinion in …, 2010 - Elsevier
Small molecules that bind to normally unoccupied thyroxine (T4) binding sites within
transthyretin (TTR) in the blood stabilize the tetrameric ground state of TTR relative to the …

Pathogenesis of and therapeutic strategies to ameliorate the transthyretin amyloidoses

Y Sekijima, JW Kelly, S Ikeda - Current pharmaceutical design, 2008 - ingentaconnect.com
Transthyretin (TTR) is a homotetrameric serum and cerebrospinal fluid protein that
transports both thyroxine (T4) and the retinol-retinol binding protein complex (holoRBP) …

Repositioning tolcapone as a potent inhibitor of transthyretin amyloidogenesis and associated cellular toxicity

R Sant'Anna, P Gallego, LZ Robinson… - Nature …, 2016 - nature.com
Transthyretin (TTR) is a plasma homotetrameric protein implicated in fatal systemic
amyloidoses. TTR tetramer dissociation precedes pathological TTR aggregation. Native …

Orally administered diflunisal stabilizes transthyretin against dissociation required for amyloidogenesis

Y Sekijima, MA Dendle, JW Kelly - Amyloid, 2006 - Taylor & Francis
Objective. Rate-limiting transthyretin (TTR) tetramer dissociation and monomer misfolding
enable misassembly into numerous aggregate morphologies including amyloid, a process …

[HTML][HTML] Natural polyphenols inhibit different steps of the process of transthyretin (TTR) amyloid fibril formation

N Ferreira, MJ Saraiva, MR Almeida - FEBS letters, 2011 - Elsevier
Several natural polyphenols with potent inhibitory effects on amyloid fibril formation have
been reported. Herein, we studied modulation of transthyretin (TTR) fibrillogenesis by …

[HTML][HTML] Binding of epigallocatechin-3-gallate to transthyretin modulates its amyloidogenicity

N Ferreira, I Cardoso, MR Domingues, R Vitorino… - FEBS letters, 2009 - Elsevier
More than 100 transthyretin (TTR) variants are associated with hereditary amyloidosis.
Approaches for TTR amyloidosis that interfere with any step of the cascade of events leading …

Undiscovered roles for transthyretin: from a transporter protein to a new therapeutic target for Alzheimer's disease

T Gião, J Saavedra, E Cotrina, J Quintana… - International journal of …, 2020 - mdpi.com
Transthyretin (TTR), an homotetrameric protein mainly synthesized by the liver and the
choroid plexus, and secreted into the blood and the cerebrospinal fluid, respectively, has …

Update on the clinical utility of an RNA interference-based treatment: focus on Patisiran

M Rizk, Ş Tüzmen - Pharmacogenomics and personalized …, 2017 - Taylor & Francis
RNA interference (RNAi) is a naturally existing endogenous mechanism for post-
transcriptional gene regulation, nowadays commonly utilized for functional characterization …

Epigallocatechin-3-gallate as a potential therapeutic drug for TTR-related amyloidosis:“in vivo” evidence from FAP mice models

N Ferreira, MJ Saraiva, MR Almeida - PloS one, 2012 - journals.plos.org
Background Familial amyloidotic polyneuropathy (FAP) is a neurodegenerative disease
caused by the extracellular deposition of mutant transthyretin (TTR), with special …

Treatment of transthyretin amyloid cardiomyopathy: the current options, the future, and the challenges

C Tschöpe, A Elsanhoury - Journal of Clinical Medicine, 2022 - mdpi.com
Transthyretin amyloid cardiomyopathy (ATTR-CM) is a progressively debilitating, rare
disease associated with high mortality. ATTR-CM occurs when TTR amyloid protein builds …