ALS-Linked Protein FUS Plays Major Role in Gene Expression, Study Finds

Scientists have discovered a key role for the ALS-linked protein known as fused in sarcoma (FUS) in a system that is crucial to regulating gene expression. The study, “FUS Regulates Activity of MicroRNA-Mediated Gene Silencing,” was published in the journal Molecular Cell. FUS is a DNA/RNA-binding protein involved in repairing…

I recently volunteered to participate in the Precision Medicine Program (PMP) of the ALS Therapy Development Institute (ALS TDI). I chose them because all of their funds go toward research, I like doing my part to help support the ALS community, and it allows me to participate…

Everyone suffers from stress from time to time but when you have a chronic disease, stress not only adds to your everyday burden but can often exacerbate the symptoms of your condition. While we can’t avoid stress altogether, there are ways that we help ourselves relax more and not let things…

Syracuse University researchers have obtained further insight into the protein UBQLN2, whose mutations have been associated with the development of amyotrophic lateral sclerosis (ALS). The study, “Ubiquitin Modulates Liquid-Liquid Phase Separation of UBQLN2 via Disruption of Multivalent Interactions,” was published in the journal Molecular Cell. There are many…

High levels of a protein that transports vitamin A through the body helps protect against the development and progression of ALS, a German study suggests. The protein, retinol binding protein 4 (RBP4), is secreted by fat tissue. The study’s findings suggest it is a potential target for a therapy that could…

In his 10 months on the job, Commissioner Scott Gottlieb of the U.S. Food and Drug Administration is earning praise for his efforts to make clinical trials for new therapies more flexible and responsive to the needs of rare disease patients. From cystic fibrosis to epidermolysis bullosa, the FDA…

The Montreal Neurological Institute of McGill University and Thermo Fisher Scientific have recently partnered to accelerate the understanding of neurological disease by focusing on about 30 proteins associated with amyotrophic lateral sclerosis (ALS) and other brain diseases. The collaboration is part of…

At a time of unprecedented polarization in Congress, two U.S. lawmakers — one Republican, one Democrat — are stressing the urgency of working across the aisle to help the estimated 30 million Americans with rare diseases. Rep. Leonard Lance (R-New Jersey) and Sen. Amy Klobuchar (D-Minnesota) spoke to more…

One of the most frustrating symptoms of living with a chronic illness is brain fog. There are medications to treat many symptoms of chronic diseases, but sadly there isn’t yet a pill that takes away brain fog. However, there are ways to deal with it so patients can minimize its effects and…

Researchers have identified genes that promote amyotrophic lateral sclerosis (ALS), using the gene-editing technology CRISPR-Cas9. The findings represent not only another piece of information to understand the molecular mechanisms triggering ALS, but also support the newly identified genes as potential targets for future therapeutics. The study “CRISPR–Cas9 screens in human cells…