ALS genetics, mechanisms, and therapeutics: where are we now?

R Mejzini, LL Flynn, IL Pitout, S Fletcher… - Frontiers in …, 2019 - frontiersin.org
The scientific landscape surrounding amyotrophic lateral sclerosis (ALS) continues to shift
as the number of genes associated with the disease risk and pathogenesis, and the cellular …

Physiological functions and pathobiology of TDP‐43 and FUS/TLS proteins

A Ratti, E Buratti - Journal of neurochemistry, 2016 - Wiley Online Library
The multiple roles played by RNA binding proteins in neurodegeneration have become
apparent following the discovery of TAR DNA binding protein 43 kDa (TDP‐43) and fused in …

ALS/FTD-linked mutation in FUS suppresses intra-axonal protein synthesis and drives disease without nuclear loss-of-function of FUS

J López-Erauskin, T Tadokoro, MW Baughn, B Myers… - Neuron, 2018 - cell.com
Through the generation of humanized FUS mice expressing full-length human FUS, we
identify that when expressed at near endogenous murine FUS levels, both wild-type and …

The role of FUS gene variants in neurodegenerative diseases

H Deng, K Gao, J Jankovic - Nature Reviews Neurology, 2014 - nature.com
The neurodegenerative diseases are a diverse group of disorders characterized by
progressive loss of specific groups of neurons. These diseases affect different populations …

Nearly 30 years of animal models to study amyotrophic lateral sclerosis: a historical overview and future perspectives

T Bonifacino, RA Zerbo, M Balbi, C Torazza… - International journal of …, 2021 - mdpi.com
Amyotrophic lateral sclerosis (ALS) is a fatal, multigenic, multifactorial, and non-cell
autonomous neurodegenerative disease characterized by upper and lower motor neuron …

ALS/FTD‐associated FUS activates GSK‐3β to disrupt the VAPB–PTPIP 51 interaction and ER–mitochondria associations

R Stoica, S Paillusson, P Gomez‐Suaga, JC Mitchell… - EMBO …, 2016 - embopress.org
Defective FUS metabolism is strongly associated with amyotrophic lateral sclerosis and
frontotemporal dementia (ALS/FTD), but the mechanisms linking FUS to disease are not …

[HTML][HTML] RNA-binding proteins in amyotrophic lateral sclerosis

M Zhao, JR Kim, R van Bruggen, J Park - Molecules and cells, 2018 - Elsevier
Significant research efforts are ongoing to elucidate the complex molecular mechanisms
underlying amyotrophic lateral sclerosis (ALS), which may in turn pinpoint potential …

Advances in treating amyotrophic lateral sclerosis: insights from pathophysiological studies

S Vucic, JD Rothstein, MC Kiernan - Trends in neurosciences, 2014 - cell.com
Amyotrophic lateral sclerosis (ALS) is the most frequently occurring of the neuromuscular
degenerative disorders, with a median survival time of 3–5 years. The pathophysiological …

[HTML][HTML] Mechanisms of FUS mutations in familial amyotrophic lateral sclerosis

Y Shang, EJ Huang - Brain research, 2016 - Elsevier
Recent advances in the genetics of amyotrophic lateral sclerosis (ALS) have provided key
mechanistic insights to the pathogenesis of this devastating neurodegenerative disease …

Mouse models of ALS: Past, present and future

C Lutz - Brain Research, 2018 - Elsevier
Genome sequencing of both sporadic and familial patients of Amyotrophic Lateral Sclerosis
(ALS) has led to the identification of new genes that are both contributing and causative in …