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AG10 inhibits amyloidogenesis and cellular toxicity of the familial amyloid cardiomyopathy-associated V122I transthyretin
The development of AG10 is reported, a potent and selective kinetic stabilizer of TTR, which prevents dissociation of V122I-TTR in serum samples obtained from patients with familial amyloid cardiomyopathy and makes it a very promising candidate to treat TTR amyloids cardiopathy. Expand
Evidence that alpha-synuclein does not inhibit phospholipase D.
It is proposed that the previously reported effects of alphaSyn on PLD activity could be due to increased endoplasmic reticulum-related stress associated with alphaSyn overexpression in cells, but are not likely due to a specific and direct interaction between alphaSyn and PLD. Expand
Lipidomic profiling in mouse brain reveals differences between ages and genders, with smaller changes associated with α‐synuclein genotype
This work applied phospholipid profiling to mouse models relevant to Parkinson’s disease, using mice that were transgenic for human α‐synuclein (αSyn) or deleted of endogenous αSyn to identify age‐related changes relevant both to Parkinson's disease and to normal aging. Expand
Quantification of Transthyretin Kinetic Stability in Human Plasma Using Subunit Exchange
Subunit exchange was used to quantify the stabilization of WT TTR from senile systemic amyloidosis patients currently being treated with tafamidis (20 mg orally, once daily), and TTR kinetic stability correlated with the tAFamidis plasma concentration. Expand
Evidence That #-Synuclein Does Not Inhibit Phospholipase D †
R-Synuclein (RSyn) is a small cytosolic protein of unknown function, which is highly enriched in the brain. It is genetically linked to Parkinson’s disease (PD) in that missense mutations orExpand