amyotrophic lateral sclerosis

[Molecular Therapy] Scientists review genome editing approaches aimed either at correcting the disease-causing mutations or at modulating the expression of genetic modifiers, e.g. by repairing SOD1 mutations or SMN2 splicing defect, or deleting C9orf72 expanded repeats.

[Ionis Pharmaceuticals, Inc.] Ionis Pharmaceuticals, Inc. announced the initiation of a Phase III clinical trial of ION363 in patients with amyotrophic lateral sclerosis (ALS) with mutations in the fused in sarcoma gene.

[Molecular Therapy-Methods & Clinical Development] Researchers tested the in vitro neuroprotective potential of mouse adipose derived stem cells in astrocyte/motor neuron co-cultures where amyotrophic lateral sclerosis (ALS) astrocytes show neurotoxicity.

[Proceedings of the National Academy of Sciences of the United States of America] The authors identified a subclass of microglia in mouse models of ALS which we term receptor-interacting protein kinase 1 (RIPK1)-Regulated Inflammatory Microglia.

[Autophagy] The authors showed elevated activation of the MTOR pathway in human-derived astrocytes harboring mutant SOD1, which resulted in inhibition of macroautophagy/autophagy, increased cell proliferation, and enhanced astrocyte reactivity.

[Proceedings of the National Academy of Sciences of the United States of America] The authors showed environmentally induced nitrosative stress triggered protein aggregation and cell-to-cell spread.