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 …
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 …
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) …
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 …
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 …
failure with preserved ejection fraction (HFpEF). Background: Left ventricular amyloid …
Protein misfolding, functional amyloid, and human disease
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 …
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 …
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
A range of human degenerative conditions, including Alzheimer's disease, light-chain
amyloidosis and the spongiform encephalopathies, is associated with the deposition in …
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 …
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 …
of strategies to control amyloid formation and overcome neurodegenerative disorders …