ALS genetics: gains, losses, and implications for future therapies

G Kim, O Gautier, E Tassoni-Tsuchida, XR Ma… - Neuron, 2020‏ - cell.com
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disorder caused by the loss
of motor neurons from the brain and spinal cord. The ALS community has made remarkable …

Liquid-liquid phase separation of TDP-43 and FUS in physiology and pathology of neurodegenerative diseases

JL Carey, L Guo - Frontiers in molecular biosciences, 2022‏ - frontiersin.org
Liquid-liquid phase separation of RNA-binding proteins mediates the formation of numerous
membraneless organelles with essential cellular function. However, aberrant phase …

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 …

Genetics of amyotrophic lateral sclerosis: A review

S Mathis, C Goizet, A Soulages, JM Vallat… - Journal of the …, 2019‏ - Elsevier
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disorder of the motor pathways,
invariably leading to death within a few years of onset. Most cases of ALS are sporadic, but …

Protein aggregation in amyotrophic lateral sclerosis

AM Blokhuis, EJN Groen, M Koppers… - Acta …, 2013‏ - Springer
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease characterized by the
aggregation of ubiquitinated proteins in affected motor neurons. Recent studies have …

Dendritic spines: the locus of structural and functional plasticity

C Sala, M Segal - Physiological reviews, 2014‏ - journals.physiology.org
The introduction of high-resolution time lapse imaging and molecular biological tools has
changed dramatically the rate of progress towards the understanding of the complex …

The tip of the iceberg: RNA-binding proteins with prion-like domains in neurodegenerative disease

OD King, AD Gitler, J Shorter - Brain research, 2012‏ - Elsevier
Prions are self-templating protein conformers that are naturally transmitted between
individuals and promote phenotypic change. In yeast, prion-encoded phenotypes can be …

RNA transport and local translation in neurodevelopmental and neurodegenerative disease

MS Fernandopulle, J Lippincott-Schwartz… - Nature …, 2021‏ - nature.com
Neurons decentralize protein synthesis from the cell body to support the active metabolism
of remote dendritic and axonal compartments. The neuronal RNA transport apparatus …

TDP-43 and FUS in amyotrophic lateral sclerosis and frontotemporal dementia

IRA Mackenzie, R Rademakers… - The Lancet Neurology, 2010‏ - thelancet.com
Abnormal intracellular protein aggregates comprise a key characteristic in most
neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS) and …

Interaction of FUS and HDAC1 regulates DNA damage response and repair in neurons

WY Wang, L Pan, SC Su, EJ Quinn, M Sasaki… - Nature …, 2013‏ - nature.com
Defects in DNA repair have been extensively linked to neurodegenerative diseases, but the
exact mechanisms remain poorly understood. We found that FUS, an RNA/DNA-binding …