Transthyretin-Related Amyloidoses: A Structural and Thermodynamic Approach
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منابع مشابه
Curcumin: A multi-target disease-modifying agent for late-stage transthyretin amyloidosis
Transthyretin amyloidoses encompass a variety of acquired and hereditary diseases triggered by systemic extracellular accumulation of toxic transthyretin aggregates and fibrils, particularly in the peripheral nervous system. Since transthyretin amyloidoses are typically complex progressive disorders, therapeutic approaches aiming multiple molecular targets simultaneously, might improve therapy ...
متن کاملTissue damage in the amyloidoses: Transthyretin monomers and nonnative oligomers are the major cytotoxic species in tissue culture.
The transthyretin (TTR) amyloidoses are human diseases in which the misfolded TTR protein aggregates in tissues with subsequent visceral, peripheral, and autonomic nerve dysfunction. Recent reports have stressed the importance of oligomeric intermediates as major cytotoxic species in various forms of amyloidogenesis. We have examined the cytotoxic effects of several quaternary structural states...
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Background—Differentiating immunoglobulin light-chain (AL) from transthyretin-related cardiac amyloidoses (ATTR) is imperative given implications for prognosis, therapy, and genetic counseling. We validated the discriminatory ability of Tc-pyrophosphate scintigraphy (TcPYP) in AL vs. TTR-related cardiac amyloidoses. Methods and Results—45 subjects (12 AL, 16 ATTR wild-type, and 17 ATTR mutants)...
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Transthyretin amyloidosis. Infectious amyloid proteins. Unconventional slow virus diseases. Spongiform encephalopathies. Genetic control and point mutations.
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Transthyretin (TTR) is a plasma homotetrameric protein implicated in fatal systemic amyloidoses. TTR tetramer dissociation precedes pathological TTR aggregation. Native state stabilizers are promising drugs to treat TTR amyloidoses. Here we repurpose tolcapone, an FDA-approved molecule for Parkinson's disease, as a potent TTR aggregation inhibitor. Tolcapone binds specifically to TTR in human p...
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