ALS genetics, mechanisms, and therapeutics: where are we now?
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 …
as the number of genes associated with the disease risk and pathogenesis, and the cellular …
The role of TDP-43 mislocalization in amyotrophic lateral sclerosis
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 …
of patients with Amyotrophic Lateral Sclerosis (ALS), TAR DNA Binding Protein 43 kDa (TDP …
The pathobiology of TDP-43 C-terminal fragments in ALS and FTLD
During neurodegenerative disease, the multifunctional RNA-binding protein TDP-43
undergoes a vast array of post-translational modifications, including phosphorylation …
undergoes a vast array of post-translational modifications, including phosphorylation …
ALS-implicated protein TDP-43 sustains levels of STMN2, a mediator of motor neuron growth and repair
The findings that amyotrophic lateral sclerosis (ALS) patients almost universally display
pathological mislocalization of the RNA-binding protein TDP-43 and that mutations in its …
pathological mislocalization of the RNA-binding protein TDP-43 and that mutations in its …
Monomerization of TDP-43 is a key determinant for inducing TDP-43 pathology in amyotrophic lateral sclerosis
K Oiwa, S Watanabe, K Onodera, Y Iguchi… - Science …, 2023 - science.org
The cytoplasmic aggregation of TAR DNA binding protein-43 (TDP-43), also known as TDP-
43 pathology, is the pathological hallmark of amyotrophic lateral sclerosis (ALS). However …
43 pathology, is the pathological hallmark of amyotrophic lateral sclerosis (ALS). However …
PIKFYVE inhibition mitigates disease in models of diverse forms of ALS
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease that results from
many diverse genetic causes. Although therapeutics specifically targeting known causal …
many diverse genetic causes. Although therapeutics specifically targeting known causal …
Autophagy and ALS: mechanistic insights and therapeutic implications
Mechanisms of protein homeostasis are crucial for overseeing the clearance of misfolded
and toxic proteins over the lifetime of an organism, thereby ensuring the health of neurons …
and toxic proteins over the lifetime of an organism, thereby ensuring the health of neurons …
[HTML][HTML] Axonal transport of TDP-43 mRNA granules is impaired by ALS-causing mutations
The RNA-binding protein TDP-43 regulates RNA metabolism at multiple levels, including
transcription, RNA splicing, and mRNA stability. TDP-43 is a major component of the …
transcription, RNA splicing, and mRNA stability. TDP-43 is a major component of the …
Protein aggregation in amyotrophic lateral sclerosis
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease characterized by the
aggregation of ubiquitinated proteins in affected motor neurons. Recent studies have …
aggregation of ubiquitinated proteins in affected motor neurons. Recent studies have …
[HTML][HTML] TDP-43 proteinopathy and ALS: insights into disease mechanisms and therapeutic targets
Therapeutic options for patients with amyotrophic lateral sclerosis (ALS) are currently
limited. However, recent studies show that almost all cases of ALS, as well as tau-negative …
limited. However, recent studies show that almost all cases of ALS, as well as tau-negative …