protein clumps

Researchers at the University of Waterloo will use a CA$125,000 (about $93,000) grant from the ALS Society of Canada and Brain Canada to better understand how proteins become misplaced in cells and build into the toxic clumps that mark amyotrophic lateral sclerosis (ALS). Led by Dale Martin, PhD,…

VectorY Therapeutics has raised about $138 million in funding aiming to bring into clinical testing VTx-002, its lead treatment candidate for amyotrophic lateral sclerosis (ALS). The therapy equips nerve cells to produce antibodies against the damaging TDP-43 protein clumps that are an ALS hallmark. “The investment…

When the TDP-43 protein is less able to form two-protein complexes called dimers, it appears to be more likely to form the toxic clumps seen in amyotrophic lateral sclerosis (ALS), a study reports. As these clumps are found in about 97% of all ALS cases, a better understanding of the…

Mutations in the FUS gene, which cause some forms of amyotrophic lateral sclerosis (ALS), result in a circular RNA molecule called circ-Hdgfrp3 being pulled into toxic protein clumps that form in nerve cells, according to a study. This is the first time that a circular RNA has been shown…

A team of scientists in the U.K. and Japan has determined the structure of aggregated TDP-43, the protein whose abnormal clumps are characteristic of amyotrophic lateral sclerosis (ALS). Their work, reportedly the first to reveal the molecular structure of aggregated TDP-43, identified a “double-spiral fold” of the protein in patients’ brain…

The loss of chemical modifications in the regulatory sequence of the TARDBP gene, called DNA methylation, seems to contribute to increased levels and clumping of the TDP-43 protein in the motor cortex of people with amyotrophic lateral sclerosis (ALS), a study found. As methylation in this gene — which…

Seelos Therapeutics’ experimental therapy SLS-005 (trehalose) will serve as the fifth arm of the multi-regimen HEALEY trial for amyotrophic lateral sclerosis (ALS), after being cleared by the U.S. Food and Drug Administration and the Mass General Brigham Institutional Review Board. With this final regulatory authorization, Seelos may still…

Seelos Therapeutics has announced plans to start patient dosing in a pivotal Phase 2b/3 trial testing its experimental therapy SLS-005 (trehalose), designed to prevent protein clumping in cells, in adults with amyotrophic lateral sclerosis (ALS) by the close of September. The study is part of the HEALEY ALS Platform…

In contrast to what is often assumed, new research suggests that clumps of the protein TDP-43 — a hallmark of amyotrophic lateral sclerosis (ALS) — actually protect nerve cells rather than harm them. If this finding confirmed in future studies, it could hugely impact how ALS and other neurodegenerative diseases…

Clumps of the protein FUS, which are a pathological characteristic of ALS and frontotemporal dementia, can be prevented through a biological process called phosphorylation, a new study shows. The study, “Phosphorylation of the FUS low‐complexity domain disrupts phase separation, aggregation, and toxicity,” was published in the EMBO Journal. Phosphorylation…