The role of TDP-43 mislocalization in amyotrophic lateral sclerosis

TR Suk, MWC Rousseaux - Molecular neurodegeneration, 2020 - Springer
Since its discovery as a primary component in cytoplasmic aggregates in post-mortem tissue
of patients with Amyotrophic Lateral Sclerosis (ALS), TAR DNA Binding Protein 43 kDa (TDP …

Amyotrophic lateral sclerosis

RH Brown, A Al-Chalabi - New England Journal of Medicine, 2017 - Mass Medical Soc
Amyotrophic Lateral Sclerosis | New England Journal of Medicine Skip to main content The
New England Journal of Medicine homepage Advanced Search SEARCH SPECIALTIES …

Therapeutic reduction of ataxin-2 extends lifespan and reduces pathology in TDP-43 mice

LA Becker, B Huang, G Bieri, R Ma, DA Knowles… - Nature, 2017 - nature.com
Amyotrophic lateral sclerosis (ALS) is a rapidly progressing neurodegenerative disease that
is characterized by motor neuron loss and that leads to paralysis and death 2–5 years after …

Human sensorimotor organoids derived from healthy and amyotrophic lateral sclerosis stem cells form neuromuscular junctions

JD Pereira, DM DuBreuil, AC Devlin, A Held… - Nature …, 2021 - nature.com
Human induced pluripotent stem cells (iPSC) hold promise for modeling diseases in
individual human genetic backgrounds and thus for developing precision medicine. Here …

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 …

Autophagy in neurodegeneration: New insights underpinning therapy for neurological diseases

O Corti, K Blomgren, A Poletti… - Journal of …, 2020 - Wiley Online Library
In autophagy long‐lived proteins, protein aggregates or damaged organelles are engulfed
by vesicles called autophagosomes prior to lysosomal degradation. Autophagy dysfunction …

Functional significance of TDP-43 mutations in disease

E Buratti - Advances in genetics, 2015 - Elsevier
At present, there are very few therapeutic options for patients affected by amyotrophic lateral
sclerosis (ALS) and frontotemporal dementia (FTD). However, almost all patients affected by …

Loss of mouse Stmn2 function causes motor neuropathy

IG San Juan, LA Nash, KS Smith, MF Leyton-Jaimes… - Neuron, 2022 - cell.com
Amyotrophic lateral sclerosis (ALS) is characterized by motor neuron degeneration
accompanied by aberrant accumulation and loss of function of the RNA-binding protein …

From animal models to human disease: a genetic approach for personalized medicine in ALS

V Picher-Martel, PN Valdmanis, PV Gould… - Acta neuropathologica …, 2016 - Springer
Abstract Amyotrophic Lateral Sclerosis (ALS) is the most frequent motor neuron disease in
adults. Classical ALS is characterized by the death of upper and lower motor neurons …

Molecular mechanisms underlying TDP-43 pathology in cellular and animal models of ALS and FTLD

A Wood, Y Gurfinkel, N Polain, W Lamont… - International journal of …, 2021 - mdpi.com
Amyotrophic lateral sclerosis (ALS) and frontotemporal lobar degeneration (FTLD) are
neurodegenerative disorders that exist on a disease spectrum due to pathological, clinical …