RPS25 helps translate repetitive snippets of RNA that are associated with neurodegenerative diseases. Knocking it down reduces protein aggregates and cell death.
Data on ASOs, presented recently at the annual meeting of the American Academy of Neurology, depict RNA-based therapies as broadly on the rise in neurodegenerative diseases.
In a new, inducible mouse model, poly(GR) damages mitochondria, but its effect is reversible. In flies, turning off transcription of hexanucleotide expansion protects cells.
Liquid beads of TDP-43 form independently of stress granules and sequester proteins needed for nucleocytoplasmic transport.
In a tiny pilot trial, people with amyotrophic lateral sclerosis who took a “cellular health and optimization” supplement were reported to have improved on several clinical outcome measures.
New work implicates changes in chromatin structure and failed DNA repair in neurodegeneration.
Two independent studies find that loss of nuclear TDP-43 leads to mis-splicing of stathmin 2, an essential protein for axon growth and repair.
In a fly model of neurodegeneration, CDK5 slams autophagy, which leads to a runaway immune response that shoves aging neurons over the edge.
Proteomics and protein-protein interaction research may yield clues to etiology, tracking, and treatment of granulin-related and other forms of FTD/ALS.
Scientists say chaperones keep FUS from joining up with aggregating proteins as FUS makes its way down axons to deliver RNAs for local translation. ALS/FTD mutations bungle the process.
At a meeting in San Diego, researchers traded news about how TDP-43 gets trapped in the cytoplasm, finding both good and bad consequences of its exodus from the nucleus.
At an RNA metabolism meeting, scientists reported connections between C9ORF72 loss and gain of function. Their talks brimmed with new biology implicating autophagy, the cellular stress response, and RAN translation off introns.
Swine that express mutated human SOD1 exhibit the hallmarks of ALS pathology, including aggregates and degeneration in motor neurons, preceded by a lengthy preclinical phase.
In mice, a daily low dose restored regulatory T cells and held off symptoms of a demyelinating disease. Could an old, over-the-counter drug help with ALS, in which similar cells decline?
Cultured sensory neurons from ALS mouse models have truncated, less-complex neurites that grow slowly compared to controls.