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 …

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 …

Antisense oligonucleotide silencing of FUS expression as a therapeutic approach in amyotrophic lateral sclerosis

VA Korobeynikov, AK Lyashchenko… - Nature medicine, 2022 - nature.com
Fused in sarcoma (FUS) is an RNA-binding protein that is genetically and pathologically
associated with rare and aggressive forms of amyotrophic lateral sclerosis (ALS) and …

Phosphorylation of the FUS low‐complexity domain disrupts phase separation, aggregation, and toxicity

Z Monahan, VH Ryan, AM Janke, KA Burke… - The EMBO …, 2017 - embopress.org
Neuronal inclusions of aggregated RNA‐binding protein fused in sarcoma (FUS) are
hallmarks of ALS and frontotemporal dementia subtypes. Intriguingly, FUS's nearly …

[HTML][HTML] The role of mitochondria in amyotrophic lateral sclerosis

EF Smith, PJ Shaw, KJ De Vos - Neuroscience letters, 2019 - Elsevier
Mitochondria are unique organelles that are essential for a variety of cellular processes
including energy metabolism, calcium homeostasis, lipid biosynthesis, and apoptosis …

Role of circular RNAs in brain development and CNS diseases

SL Mehta, RJ Dempsey, R Vemuganti - Progress in neurobiology, 2020 - Elsevier
In mammals, many classes of noncoding RNAs (ncRNAs) are expressed at a much higher
level in the brain than in other organs. Recent studies have identified a new class of ncRNAs …

RNA buffers the phase separation behavior of prion-like RNA binding proteins

S Maharana, J Wang, DK Papadopoulos, D Richter… - Science, 2018 - science.org
Prion-like RNA binding proteins (RBPs) such as TDP43 and FUS are largely soluble in the
nucleus but form solid pathological aggregates when mislocalized to the cytoplasm. What …

The bowel and beyond: the enteric nervous system in neurological disorders

M Rao, MD Gershon - Nature reviews Gastroenterology & hepatology, 2016 - nature.com
The enteric nervous system (ENS) is large, complex and uniquely able to orchestrate
gastrointestinal behaviour independently of the central nervous system (CNS). An intact …

FUS affects circular RNA expression in murine embryonic stem cell-derived motor neurons

L Errichelli, S Dini Modigliani, P Laneve… - Nature …, 2017 - nature.com
The RNA-binding protein FUS participates in several RNA biosynthetic processes and has
been linked to the pathogenesis of amyotrophic lateral sclerosis (ALS) and frontotemporal …

HDAC6 inhibition reverses axonal transport defects in motor neurons derived from FUS-ALS patients

W Guo, M Naujock, L Fumagalli, T Vandoorne… - Nature …, 2017 - nature.com
Amyotrophic lateral sclerosis (ALS) is a rapidly progressive neurodegenerative disorder due
to selective loss of motor neurons (MNs). Mutations in the fused in sarcoma (FUS) gene can …