[HTML][HTML] 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 …

[HTML][HTML] New developments and opportunities in drugs being trialed for amyotrophic lateral sclerosis from 2020 to 2022

JS Jiang, Y Wang, M Deng - Frontiers in pharmacology, 2022 - frontiersin.org
Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disorder that
primarily affects motor neurons in the brain and spinal cord. In the recent past, there have …

[HTML][HTML] ELAVL4, splicing, and glutamatergic dysfunction precede neuron loss in MAPT mutation cerebral organoids

KR Bowles, MC Silva, K Whitney, T Bertucci, JE Berlind… - Cell, 2021 - cell.com
Frontotemporal dementia (FTD) because of MAPT mutation causes pathological
accumulation of tau and glutamatergic cortical neuronal death by unknown mechanisms. We …

PIKFYVE inhibition mitigates disease in models of diverse forms of ALS

ST Hung, GR Linares, WH Chang, Y Eoh, G Krishnan… - Cell, 2023 - cell.com
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease that results from
many diverse genetic causes. Although therapeutics specifically targeting known causal …

[HTML][HTML] Molecular and cellular mechanisms affected in ALS

L Le Gall, E Anakor, O Connolly… - Journal of personalized …, 2020 - mdpi.com
Amyotrophic lateral sclerosis (ALS) is a terminal late-onset condition characterized by the
loss of upper and lower motor neurons. Mutations in more than 30 genes are associated to …

[HTML][HTML] Human stem cell models of neurodegeneration: From basic science of amyotrophic lateral sclerosis to clinical translation

E Giacomelli, BF Vahsen, EL Calder, Y Xu, J Scaber… - Cell Stem Cell, 2022 - cell.com
Neurodegenerative diseases are characterized by progressive cell loss leading to disruption
of the structure and function of the central nervous system. Amyotrophic lateral sclerosis …

[HTML][HTML] Gene therapy in amyotrophic lateral sclerosis

T Fang, G Je, P Pacut, K Keyhanian, J Gao, M Ghasemi - Cells, 2022 - mdpi.com
Since the discovery of Cu/Zn superoxide dismutase (SOD1) gene mutation, in 1993, as the
first genetic abnormality in amyotrophic lateral sclerosis (ALS), over 50 genes have been …

[HTML][HTML] Emerging therapies and novel targets for TDP-43 proteinopathy in ALS/FTD

LR Hayes, P Kalab - Neurotherapeutics, 2022 - Elsevier
Nuclear clearance and cytoplasmic mislocalization of the essential RNA binding protein,
TDP-43, is a pathologic hallmark of amyotrophic lateral sclerosis, frontotemporal dementia …

SYF2 suppression mitigates neurodegeneration in models of diverse forms of ALS

GR Linares, Y Li, WH Chang, J Rubin-Sigler… - Cell Stem Cell, 2023 - cell.com
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease caused by many
diverse genetic etiologies. Although therapeutics that specifically target causal mutations …

Blood-CNS barrier dysfunction in amyotrophic lateral sclerosis: proposed mechanisms and clinical implications

M Steinruecke, RM Lonergan… - Journal of Cerebral …, 2023 - journals.sagepub.com
There is strong evidence for blood-brain and blood-spinal cord barrier dysfunction at the
early stages of many neurodegenerative diseases, including amyotrophic lateral sclerosis …