Results 111 to 120 of about 45,139 (271)

Repositioning tolcapone as a potent inhibitor of transthyretin amyloidogenesis and associated cellular toxicity

open access: yesNature Communications, 2016
Misfolding of transthyretin can cause amyloid aggregation disorders that can be treated by stabilizing the tetrameric form with tafamidis. Here the authors show that tolcapone, a drug already FDA-approved for Parkinson disease, has strong transthyretin ...
Ricardo Sant'Anna   +15 more
doaj   +1 more source

A Comprehensive Review on Capillary Electrophoresis–Mass Spectrometry in Advancing Biomolecular Research

open access: yesELECTROPHORESIS, EarlyView.
ABSTRACT This review provides an in‐depth exploration of capillary electrophoresis–mass spectrometry (CE–MS) in biomolecular research from 2020 to 2024. CE–MS emerges as a versatile and powerful tool due to its numerous advantages, facilitating the analysis of various biomolecules, including proteins, peptides, oligonucleotides, and other metabolites ...
Laura Pont   +2 more
wiley   +1 more source

Real-life experience with inotersen at CEPARM, Hospital Universitário Clementino Fraga Filho, Universidade Federal do Rio de Janeiro

open access: yesArquivos de Neuro-Psiquiatria
Background Hereditary transthyretin amyloidosis (ATTRv) is an inherited, progressive, and fatal disease still largely underdiagnosed. Mutations in the transthyretin (TTR) gene cause the TTR protein to destabilize, misfold, aggregate, and ...
Moises Dias   +9 more
doaj   +1 more source

Exploring the Potential and Advancements of Circular RNA Therapeutics

open access: yesExploration, EarlyView.
Given the remarkable advantages in terms of stability, sustained expression profile, safety, wide range of druggable targets, scalable and cost‐effective manufacturing capabilities, circRNA is currently undergoing intensive investigation for various therapeutic applications such as vaccines, protein replacement, genetic disease treatment, gene therapy,
Lei Wang   +6 more
wiley   +1 more source

Familial amyloidosis with polyneuropathy type 1 caused by transthyretin mutation Val50Met (Val30Met): 4 cases in a non-endemic area [PDF]

open access: yes, 2018
Introduction: Transthyretin-related familial amyloid polyneuropathy (TTR-FAP) typically arises as an autonomic neuropathy primarily affecting small fibres and it occurs in adult patients in their second or third decades of life. It progresses rapidly and
Andrés, N.   +2 more
core   +2 more sources

Identification of a novel mechanism of blood-brain communication during peripheral inflammation via choroid plexus-derived extracellular vesicles [PDF]

open access: yes, 2016
Here, we identified release of extracellular vesicles (EVs) by the choroid plexus epithelium (CPE) as a new mechanism of blood-brain communication. Systemic inflammation induced an increase in EVs and associated pro-inflammatory miRNAs, including miR ...
An Hendrix   +14 more
core   +2 more sources

Palinacousis in amyloidosis: exploring the hallucinatory phenomenon in brain pathology—a case report

open access: yesJournal of Medical Case Reports
Background Hereditary transthyretin amyloidosis, caused by transthyretin gene mutations, progresses with systemic impact and often presents peripheral neuropathy.
João Martins-Correia, Luísa Sousa
doaj   +1 more source

Early Progression of Aortic Stenosis Associated With Iatrogenic Variant Transthyretin Amyloidosis After Domino Liver Transplantation

open access: yesJACC: Case Reports, 2020
We report a 65-year-old man who underwent aortic valve replacement because of severe aortic stenosis associated with de novo iatrogenic variant transthyretin amyloidosis derived from a liver graft extracted from a patient with hereditary transthyretin ...
Yasuhito Hosoda, MD   +5 more
doaj  

The Retinol Circulating Complex Releases Hormonal Ligands During Acute Stress Disorders

open access: yesFrontiers in Endocrinology, 2018
Intensive care workers actively participate in very hot debates aiming at defining the true metabolic, hormonal and nutritional requirements of critically ill patients, the contributory roles played by thyroid and retinoid ligands being largely ...
Yves Ingenbleek
doaj   +1 more source

Peripheral Nervous System Involvement of Hereditary Transthyretin Amyloidosis in the United States: A Multi‐Center Perspective

open access: yesMuscle &Nerve, EarlyView.
ABSTRACT Introduction/Aims Hereditary transthyretin amyloidosis (ATTRv) is an autosomal dominant multisystem disorder that occurs worldwide. The most common mutation in the United States, V142I, has previously been described as having a primarily cardiac presentation.
Urvi Desai   +3 more
wiley   +1 more source

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