Gene therapy for ALS: A review
DA Amado, BL Davidson - Molecular Therapy, 2021 - cell.com
Amyotrophic lateral sclerosis (ALS) has historically posed unique challenges for gene-
therapy-based approaches, due to a paucity of therapeutic targets as well as the difficulty of …
therapy-based approaches, due to a paucity of therapeutic targets as well as the difficulty of …
Polyglutamine repeats in neurodegenerative diseases
AP Lieberman, VG Shakkottai… - Annual Review of …, 2019 - annualreviews.org
Among the age-dependent protein aggregation disorders, nine neurodegenerative diseases
are caused by expansions of CAG repeats encoding polyglutamine (polyQ) tracts. We …
are caused by expansions of CAG repeats encoding polyglutamine (polyQ) tracts. We …
The changing scene of amyotrophic lateral sclerosis
W Robberecht, T Philips - Nature Reviews Neuroscience, 2013 - nature.com
Several recent breakthroughs have provided notable insights into the pathogenesis of
amyotrophic lateral sclerosis (ALS), with some even shifting our thinking about this …
amyotrophic lateral sclerosis (ALS), with some even shifting our thinking about this …
Clinical genetics of amyotrophic lateral sclerosis: what do we really know?
PM Andersen, A Al-Chalabi - Nature Reviews Neurology, 2011 - nature.com
Hereditary amyotrophic lateral sclerosis (ALS) encompasses a group of genetic disorders
characterized by adult-onset loss of the lower and upper motor neuron systems, often with …
characterized by adult-onset loss of the lower and upper motor neuron systems, often with …
Motor neuron susceptibility in ALS/FTD
AMG Ragagnin, S Shadfar, M Vidal… - Frontiers in …, 2019 - frontiersin.org
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease characterized by the
death of both upper and lower motor neurons (MNs) in the brain, brainstem and spinal cord …
death of both upper and lower motor neurons (MNs) in the brain, brainstem and spinal cord …
Genetics of amyotrophic lateral sclerosis
Amyotrophic lateral sclerosis (ALS) is a devastating, uniformly lethal degenerative disorder
of motor neurons that overlaps clinically with frontotemporal dementia (FTD). Investigations …
of motor neurons that overlaps clinically with frontotemporal dementia (FTD). Investigations …
Clinical features, neurogenetics and neuropathology of the polyglutamine spinocerebellar ataxias type 1, 2, 3, 6 and 7
U Rüb, L Schöls, H Paulson, G Auburger… - Progress in …, 2013 - Elsevier
The spinocerebellar ataxias type 1 (SCA1), 2 (SCA2), 3 (SCA3), 6 (SCA6) and 7 (SCA7) are
genetically defined autosomal dominantly inherited progressive cerebellar ataxias (ADCAs) …
genetically defined autosomal dominantly inherited progressive cerebellar ataxias (ADCAs) …
ALS: A bucket of genes, environment, metabolism and unknown ingredients
M Zufiria, FJ Gil-Bea, R Fernandez-Torron… - Progress in …, 2016 - Elsevier
The scientific scenario of amyotrophic lateral sclerosis (ALS) has dramatically changed
since TDP-43 aggregates were discovered in 2006 as the main component of the neuronal …
since TDP-43 aggregates were discovered in 2006 as the main component of the neuronal …
[HTML][HTML] RNA-binding proteins in amyotrophic lateral sclerosis
M Zhao, JR Kim, R van Bruggen, J Park - Molecules and cells, 2018 - Elsevier
Significant research efforts are ongoing to elucidate the complex molecular mechanisms
underlying amyotrophic lateral sclerosis (ALS), which may in turn pinpoint potential …
underlying amyotrophic lateral sclerosis (ALS), which may in turn pinpoint potential …
Local RNA translation at the synapse and in disease
L Liu-Yesucevitz, GJ Bassell, AD Gitler… - Journal of …, 2011 - Soc Neuroscience
Local regulation of protein synthesis in neurons has emerged as a leading research focus
because of its importance in synaptic plasticity and neurological diseases. The complexity of …
because of its importance in synaptic plasticity and neurological diseases. The complexity of …