Transthyretin (TTR) is a representative amyloidogenic protein in humans. Rate-limiting tetramer dissociation and rapid monomer misfolding and misassembly of variant TTR result in autosomal dominant familial amyloidosis. Analogous misfolding of wild-type TTR results in senile systemic amyloidosis (SSA) presenting as sporadic amyloid disease in the elderly. The objective of this review is to summarize recent progress in our understanding and treatment of TTR amyloidosis. Literature searches were conducted on the topics of transthyretin, familial amyloid polyneuropathy and clinical trials, using PubMed, the United States clinical trials directory, pharmaceutical company websites and news reports. The information was collected, evaluated for relevance and quality, critically assessed and summarized. The current standard first-line treatment of familial TTR amyloidosis is liver transplantation. However, large numbers of patients are not suitable transplant candidates. Recently, the clinical effects of TTR tetramer stabilizers, tafamidis and diflunisal, were demonstrated in randomized clinical trials, and tafamidis has been approved for the treatment of FAP in European countries and Japan. In addition, gene therapies with antisense o...Continue Reading
Interactions of retinol with binding proteins: studies with retinol-binding protein and with transthyretin
Biochemical effect of liver transplantation in two Swedish patients with familial amyloidotic polyneuropathy (FAP-met30)
The fatty acid composition of glycerolipids in nerve, brain, and other tissues of the streptozotocin diabetic rat
Strjcture of human plasma prealbumin at 2-5 A resolution. A preliminary report on the polypeptide chain conformation, quaternary structure and thyroxine binding
Liver transplantation in familial amyloidotic polyneuropathy. Follow-up of the first 20 Swedish patients
Change in variant transthyretin levels in patients with familial amyloidotic polyneuropathy type I following liver transplantation
Geographical distribution of TTR met30 carriers in northern Sweden: discrepancy between carrier frequency and prevalence rate
Clinical improvement and amyloid regression after liver transplantation in hereditary transthyretin amyloidosis
Transthyretin mutation Leu-55-Pro significantly alters tetramer stability and increases amyloidogenicity
Disruption of the transthyretin gene results in mice with depressed levels of plasma retinol and thyroid hormone
The acid-mediated denaturation pathway of transthyretin yields a conformational intermediate that can self-assemble into amyloid
Variant-sequence transthyretin (isoleucine 122) in late-onset cardiac amyloidosis in black Americans
The amyloidogenic potential of transthyretin variants correlates with their tendency to aggregate in solution
Progressive cardiac amyloidosis following liver transplantation for familial amyloid polyneuropathy: implications for amyloid fibrillogenesis
The tetrameric protein transthyretin dissociates to a non-native monomer in solution. A novel model for amyloidogenesis.
Cardiac amyloid in patients with familial amyloid polyneuropathy consists of abundant wild-type transthyretin
Suppression of transthyretin expression by ribozymes: a possible therapy for familial amyloidotic polyneuropathy
Transthyretin slowly exchanges subunits under physiological conditions: A convenient chromatographic method to study subunit exchange in oligomeric proteins
Support for the multigenic hypothesis of amyloidosis: the binding stoichiometry of retinol-binding protein, vitamin A, and thyroid hormone influences transthyretin amyloidogenicity in vitro
Energetic characteristics of the new transthyretin variant A25T may explain its atypical central nervous system pathology
Hydration and packing are crucial to amyloidogenesis as revealed by pressure studies on transthyretin variants that either protect or worsen amyloid disease
Effects of tafamidis treatment on transthyretin (TTR) stabilization, efficacy, and safety in Japanese patients with familial amyloid polyneuropathy (TTR-FAP) with Val30Met and non-Val30Met: A phase III, open-label study
Neuroinflammation in the peripheral nerve: Cause, modulator, or bystander in peripheral neuropathies?
Tuning transthyretin amyloidosis inhibition properties of iododiflunisal by combinatorial engineering of the nonsalicylic ring substitutions
Quantification of quaternary structure stability in aggregation-prone proteins under physiological conditions: the transthyretin case
Transthyretin (ATTR) amyloidosis: clinical spectrum, molecular pathogenesis and disease-modifying treatments
Substoichiometric inhibition of transthyretin misfolding by immune-targeting sparsely populated misfolding intermediates: a potential diagnostic and therapeutic for TTR amyloidoses
Transthyretin-type cerebral amyloid angiopathy: a serious complication in post-transplant patients with familial amyloid polyneuropathy
Elderly onset vitreous opacities as the initial manifestation in hereditary transthyretin (ATTR Val30Met) carries
Transthyretin cardiac amyloidosis: pathogenesis, treatments, and emerging role in heart failure with preserved ejection fraction
Monitoring treatment response to tafamidis by serial native T1 and extracellular volume in transthyretin amyloid cardiomyopathy
Diagnosis and management of transthyretin familial amyloid polyneuropathy in Japan: red-flag symptom clusters and treatment algorithm
Leading RNA Interference Therapeutics Part 1: Silencing Hereditary Transthyretin Amyloidosis, with a Focus on Patisiran.
A phase II, open-label, extension study of long-term patisiran treatment in patients with hereditary transthyretin-mediated (hATTR) amyloidosis.
Isolated cardiac amyloidosis. Utility of bone seeking tracers scintigraphy in differentiating the subtype of amyloid: A case report
Transthyretin Amyloid Fibril Disrupting Activities of Extracts and Fractions from Juglans mandshurica Maxim. var. cordiformis (Makino) Kitam
Insights on the Interaction between Transthyretin and Aβ in Solution. A Saturation Transfer Difference (STD) NMR Analysis of the Role of Iododiflunisal
Fluorotryptophan Incorporation Modulates the Structure and Stability of Transthyretin in a Site-Specific Manner
Expert opinion on monitoring symptomatic hereditary transthyretin-mediated amyloidosis and assessment of disease progression.
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