Amyotrophic lateral sclerosis: a neurodegenerative disorder poised for successful therapeutic translation
Amyotrophic lateral sclerosis (ALS) is a devastating disease caused by degeneration of
motor neurons. As with all major neurodegenerative disorders, development of disease …
motor neurons. As with all major neurodegenerative disorders, development of disease …
Molecular mechanisms underlying nucleotide repeat expansion disorders
The human genome contains over one million short tandem repeats. Expansion of a subset
of these repeat tracts underlies over fifty human disorders, including common genetic …
of these repeat tracts underlies over fifty human disorders, including common genetic …
C9orf72-mediated ALS and FTD: multiple pathways to disease
R Balendra, AM Isaacs - Nature Reviews Neurology, 2018 - nature.com
The discovery that repeat expansions in the C9orf72 gene are a frequent cause of
amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) has revolutionized …
amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) has revolutionized …
Genetics of amyotrophic lateral sclerosis: seeking therapeutic targets in the era of gene therapy
N Suzuki, A Nishiyama, H Warita, M Aoki - Journal of human genetics, 2023 - nature.com
Amyotrophic lateral sclerosis (ALS) is an intractable disease that causes respiratory failure
leading to mortality. The main locus of ALS is motor neurons. The success of antisense …
leading to mortality. The main locus of ALS is motor neurons. The success of antisense …
Non-neuronal cells in amyotrophic lateral sclerosis—from pathogenesis to biomarkers
The prevailing motor neuron-centric view of amyotrophic lateral sclerosis (ALS)
pathogenesis could be an important factor in the failure to identify disease-modifying therapy …
pathogenesis could be an important factor in the failure to identify disease-modifying therapy …
Amyotrophic lateral sclerosis
Amyotrophic lateral sclerosis (ALS), also known as motor neuron disease, is characterized
by the degeneration of both upper and lower motor neurons, which leads to muscle …
by the degeneration of both upper and lower motor neurons, which leads to muscle …
The master energy homeostasis regulator PGC-1α exhibits an mRNA nuclear export function
PGC-1α plays a central role in maintaining mitochondrial and energy metabolism
homeostasis, linking external stimuli to transcriptional co-activation of genes involved in …
homeostasis, linking external stimuli to transcriptional co-activation of genes involved in …
RAN translation at C9orf72-associated repeat expansions is selectively enhanced by the integrated stress response
Repeat-associated non-AUG (RAN) translation allows for unconventional initiation at
disease-causing repeat expansions. As RAN translation contributes to pathogenesis in …
disease-causing repeat expansions. As RAN translation contributes to pathogenesis in …
[HTML][HTML] Human stem cell models of neurodegeneration: From basic science of amyotrophic lateral sclerosis to clinical translation
Neurodegenerative diseases are characterized by progressive cell loss leading to disruption
of the structure and function of the central nervous system. Amyotrophic lateral sclerosis …
of the structure and function of the central nervous system. Amyotrophic lateral sclerosis …
Micro-RNAs secreted through astrocyte-derived extracellular vesicles cause neuronal network degeneration in C9orf72 ALS
A Varcianna, MA Myszczynska, LM Castelli, B O'Neill… - …, 2019 - thelancet.com
Background Astrocytes regulate neuronal function, synaptic formation and maintenance
partly through secreted extracellular vesicles (EVs). In amyotrophic lateral sclerosis (ALS) …
partly through secreted extracellular vesicles (EVs). In amyotrophic lateral sclerosis (ALS) …