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 …
Induced pluripotent stem cell technology: a decade of progress
Since the advent of induced pluripotent stem cell (iPSC) technology a decade ago,
enormous progress has been made in stem cell biology and regenerative medicine. Human …
enormous progress has been made in stem cell biology and regenerative medicine. Human …
TDP-43 triggers mitochondrial DNA release via mPTP to activate cGAS/STING in ALS
Cytoplasmic accumulation of TDP-43 is a disease hallmark for many cases of amyotrophic
lateral sclerosis (ALS), associated with a neuroinflammatory cytokine profile related to …
lateral sclerosis (ALS), associated with a neuroinflammatory cytokine profile related to …
Modeling sporadic ALS in iPSC-derived motor neurons identifies a potential therapeutic agent
K Fujimori, M Ishikawa, A Otomo, N Atsuta… - Nature medicine, 2018 - nature.com
Amyotrophic lateral sclerosis (ALS) is a heterogeneous motor neuron disease for which no
effective treatment is available, despite decades of research into SOD1-mutant familial ALS …
effective treatment is available, despite decades of research into SOD1-mutant familial ALS …
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 …
Microphysiological 3D model of amyotrophic lateral sclerosis (ALS) from human iPS-derived muscle cells and optogenetic motor neurons
Amyotrophic lateral sclerosis (ALS), a progressive neurodegenerative disease involving loss
of motor neurons (MNs) and muscle atrophy, still has no effective treatment, despite much …
of motor neurons (MNs) and muscle atrophy, still has no effective treatment, despite much …
Induced pluripotent stem cells and their use in human models of disease and development
The discovery of somatic cell nuclear transfer proved that somatic cells can carry the same
genetic code as the zygote, and that activating parts of this code are sufficient to reprogram …
genetic code as the zygote, and that activating parts of this code are sufficient to reprogram …
Pluripotent stem cells in disease modelling and drug discovery
Experimental modelling of human disorders enables the definition of the cellular and
molecular mechanisms underlying diseases and the development of therapies for treating …
molecular mechanisms underlying diseases and the development of therapies for treating …
STING trafficking as a new dimension of immune signaling
The cGAS–STING pathway is an evolutionarily conserved immune signaling pathway critical
for microbial defense. Unlike other innate immune pathways that largely rely on stationary …
for microbial defense. Unlike other innate immune pathways that largely rely on stationary …
Mitochondrial bioenergetic deficits in C9orf72 amyotrophic lateral sclerosis motor neurons cause dysfunctional axonal homeostasis
Axonal dysfunction is a common phenotype in neurodegenerative disorders, including in
amyotrophic lateral sclerosis (ALS), where the key pathological cell-type, the motor neuron …
amyotrophic lateral sclerosis (ALS), where the key pathological cell-type, the motor neuron …