Induced pluripotent stem cells‐based disease modeling, drug screening, clinical trials, and reverse translational research for amyotrophic lateral sclerosis

H Okano, S Morimoto, C Kato… - Journal of …, 2023 - Wiley Online Library
It has been more than 10 years since the hopes for disease modeling and drug discovery
using induced pluripotent stem cell (iPSC) technology boomed. Recently, clinical trials have …

Update on recent advances in amyotrophic lateral sclerosis

N Riva, T Domi, L Pozzi, C Lunetta, P Schito… - Journal of …, 2024 - Springer
In the last few years, our understanding of disease molecular mechanisms underpinning
ALS has advanced greatly, allowing the first steps in translating into clinical practice novel …

Mis-localization of endogenous TDP-43 leads to ALS-like early-stage metabolic dysfunction and progressive motor deficits

Y Hu, A Hruscha, C Pan, M Schifferer… - Molecular …, 2024 - Springer
Background The key pathological signature of ALS/FTLD is the mis-localization of
endogenous TDP-43 from the nucleus to the cytoplasm. However, TDP-43 gain of function in …

Variation in resting metabolic rate affects identification of metabolic change in geographically distinct cohorts of patients with ALS

CJ Holdom, MR Janse van Mantgem, J He, SL Howe… - Neurology, 2024 - AAN Enterprises
Background and Objectives Altered metabolism is observed in amyotrophic lateral sclerosis
(ALS). However, without a standardized methodology to define metabolic changes, our …

Blood–CSF barrier integrity in amyotrophic lateral sclerosis

V Klose, S Jesse, J Lewerenz, J Kassubek, J Dorst… - Brain, 2024 - academic.oup.com
The integrity of the blood-CSF barrier plays a major role in inflammation, but also in
shielding the central nervous system from external and systemic–potentially toxic–factors …

Trimetazidine Improves Mitochondrial Dysfunction in SOD1G93A Cellular Models of Amyotrophic Lateral Sclerosis through Autophagy Activation

I Salvatori, V Nesci, A Spalloni, V Marabitti… - International Journal of …, 2024 - mdpi.com
Amyotrophic Lateral Sclerosis (ALS) is considered the prototype of motor neuron disease,
characterized by motor neuron loss and muscle waste. A well-established pathogenic …

Genome-wide DNA methylation analysis related to ALS patient progression and survival

T Yang, C Li, Q Wei, D Pang, Y Cheng, J Huang… - Journal of …, 2024 - Springer
Background Epigenetics contributes to the pathogenesis of amyotrophic lateral sclerosis
(ALS). We aimed to characterize the DNA methylation profiles associated with clinical …

Volume loss in the left anterior‐superior subunit of the hypothalamus in amyotrophic lateral sclerosis

S Ghaderi, F Fatehi, S Kalra… - CNS Neuroscience …, 2024 - Wiley Online Library
Abstract Background and Objective Amyotrophic lateral sclerosis (ALS) causes motor
neuron loss and progressive paralysis. While traditionally viewed as motor neuron disease …

Non-motor symptoms in patients with amyotrophic lateral sclerosis: current state and future directions

B Bjelica, MB Bartels, J Hesebeck-Brinckmann… - Journal of …, 2024 - Springer
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease characterized by
the progressive degeneration of both upper and lower motor neurons. A defining …

Ideal body weight-based determination of minimum oral calories beneficial to function and survival in ALS

R Nakamura, M Kurihara, S Kobashi, Y Tamaki… - Frontiers in …, 2023 - frontiersin.org
Introduction This study sought to identify the optimal caloric intake to improve function and
survival in ALS patients by comparing oral intake per ideal body weight (IBW) and its …