Machado–Joseph disease/spinocerebellar ataxia type 3: lessons from disease pathogenesis and clues into therapy

CA Matos, LP de Almeida… - Journal of …, 2019 - Wiley Online Library
Abstract Machado–Joseph disease (MJD), also known as spinocerebellar ataxia type 3
(SCA 3), is an incurable disorder, widely regarded as the most common form of …

The integrated stress response and phosphorylated eukaryotic initiation factor 2α in neurodegeneration

S Bond, C Lopez-Lloreda, PJ Gannon… - … of Neuropathology & …, 2020 - academic.oup.com
The proposed molecular mechanisms underlying neurodegenerative pathogenesis are
varied, precluding the development of effective therapies for these increasingly prevalent …

UNC-49 is a redox-sensitive GABAA receptor that regulates the mitochondrial unfolded protein response cell nonautonomously

F Pohl, AL Germann, J Mao, S Hou, B Bakare… - Science …, 2023 - science.org
The γ-aminobutyric acid–mediated (GABAergic) system participates in many aspects of
organismal physiology and disease, including proteostasis, neuronal dysfunction, and life …

[HTML][HTML] Emerging topics in C. elegans aging research: Transcriptional regulation, stress response and epigenetics

MS Denzel, LR Lapierre, HID Mack - Mechanisms of ageing and …, 2019 - Elsevier
Key discoveries in aging research have been made possible with the use of model
organisms. Caenorhabditis elegans is a short-lived nematode that has become a well …

[HTML][HTML] From pathogenesis to novel therapeutics for spinocerebellar ataxia type 3: evading potholes on the way to translation

JD Da Silva, A Teixeira-Castro, P Maciel - Neurotherapeutics, 2019 - Elsevier
Abstract Spinocerebellar ataxia type 3 (SCA3), also known as Machado–Joseph disease
(MJD), is a neurodegenerative disorder caused by a polyglutamine expansion in the ATXN3 …

Consensus paper: strengths and weaknesses of animal models of spinocerebellar ataxias and their clinical implications

J Cendelin, M Cvetanovic, M Gandelman, H Hirai… - The Cerebellum, 2022 - Springer
Spinocerebellar ataxias (SCAs) represent a large group of hereditary degenerative diseases
of the nervous system, in particular the cerebellum, and other systems that manifest with a …

NPTX1 mutations trigger endoplasmic reticulum stress and cause autosomal dominant cerebellar ataxia

M Coutelier, M Jacoupy, A Janer, F Renaud, N Auger… - Brain, 2022 - academic.oup.com
With more than 40 causative genes identified so far, autosomal dominant cerebellar ataxias
exhibit a remarkable genetic heterogeneity. Yet, half the patients are lacking a molecular …

Exploring the potential of small molecule-based therapeutic approaches for targeting trinucleotide repeat disorders

AK Verma, E Khan, SR Bhagwat, A Kumar - Molecular Neurobiology, 2020 - Springer
In recent years, neurological disorders have globally become a leading cause of disability
and death. Neurological disorders are very common in both high-and low-income countries …

The mechano-ubiquitinome of articular cartilage: differential ubiquitination and activation of a group of ER-associated DUBs and ER stress regulators

N Kaokhum, A Pinto-Fernandez, M Wilkinson… - Molecular & Cellular …, 2022 - ASBMB
Understanding how connective tissue cells respond to mechanical stimulation is important to
human health and disease processes in musculoskeletal diseases. Injury to articular …

Advances in cerebellar disorders: pre-clinical models, therapeutic targets, and challenges

M Manto, J Cendelin, M Strupp… - Expert Opinion on …, 2023 - Taylor & Francis
ABSTRACT Introduction Cerebellar ataxias (CAs) represent neurological disorders with
multiple etiologies and a high phenotypic variability. Despite progress in the understanding …