Targeting protein aggregation for the treatment of degenerative diseases

YS Eisele, C Monteiro, C Fearns… - Nature reviews Drug …, 2015 - nature.com
The aggregation of specific proteins is hypothesized to underlie several degenerative
diseases, which are collectively known as amyloid disorders. However, the mechanistic …

Transthyretin (TTR) cardiac amyloidosis

FL Ruberg, JL Berk - Circulation, 2012 - Am Heart Assoc
The systemic amyloidoses are a family of diseases induced by misfolded or misassembled
proteins. Extracellular deposition of these proteins as soluble or insoluble cross-sheets …

Tafamidis for transthyretin familial amyloid polyneuropathy: a randomized, controlled trial

T Coelho, LF Maia, A Martins da Silva… - Neurology, 2012 - AAN Enterprises
Objectives: To evaluate the efficacy and safety of 18 months of tafamidis treatment in
patients with early-stage V30M transthyretin familial amyloid polyneuropathy (TTR-FAP) …

Tafamidis, a potent and selective transthyretin kinetic stabilizer that inhibits the amyloid cascade

CE Bulawa, S Connelly, M DeVit… - Proceedings of the …, 2012 - National Acad Sciences
The transthyretin amyloidoses (ATTR) are invariably fatal diseases characterized by
progressive neuropathy and/or cardiomyopathy. ATTR are caused by aggregation of …

Left ventricular amyloid deposition in patients with heart failure and preserved ejection fraction

SF Mohammed, SA Mirzoyev, WD Edwards, A Dogan… - JACC: Heart Failure, 2014 - jacc.org
Objectives: This study sought to determine the frequency of left ventricular amyloid in heart
failure with preserved ejection fraction (HFpEF). Background: Left ventricular amyloid …

Protein misfolding, functional amyloid, and human disease

F Chiti, CM Dobson - Annu. Rev. Biochem., 2006 - annualreviews.org
Peptides or proteins convert under some conditions from their soluble forms into highly
ordered fibrillar aggregates. Such transitions can give rise to pathological conditions ranging …

Molecular mechanisms of amyloidosis

G Merlini, V Bellotti - New England Journal of Medicine, 2003 - Mass Medical Soc
Amyloidosis affects millions of people, as a cause of Alzheimer's disease or a complication
of dialysis, and also causes rare conditions. The many forms of the disorder have one …

Inherent toxicity of aggregates implies a common mechanism for protein misfolding diseases

M Bucciantini, E Giannoni, F Chiti, F Baroni, L Formigli… - nature, 2002 - nature.com
A range of human degenerative conditions, including Alzheimer's disease, light-chain
amyloidosis and the spongiform encephalopathies, is associated with the deposition in …

Protein aggregation and aggregate toxicity: new insights into protein folding, misfolding diseases and biological evolution

M Stefani, CM Dobson - Journal of molecular medicine, 2003 - Springer
The deposition of proteins in the form of amyloid fibrils and plaques is the characteristic
feature of more than 20 degenerative conditions affecting either the central nervous system …

Binding mode of Thioflavin T and other molecular probes in the context of amyloid fibrils—current status

M Groenning - Journal of chemical biology, 2010 - Springer
Because understanding amyloid fibrillation in molecular detail is essential for development
of strategies to control amyloid formation and overcome neurodegenerative disorders …