UBQLN2 Mouse Models Replicate Key Human ALS Disease Features

Your research donations make a difference! ALS Association funded Dr. Mervyn Monteiro, Professor at the University of Maryland School of Medicine, developed novel ALS mouse models expressing mutant ubiquilin 2 (UBQLN2), a cause of inherited ALS-FTD that was published this week in the journal Proceedings of the National Academy of Sciences (PNAS). Importantly, these mouse models demonstrate many characteristics of ALS observed in humans, thereby providing valuable tools for identifying disease pathways in ALS and for investigating therapeutic strategies to treat ALS.

Novel Potential Therapeutic Targeting C9orf72 ALS Discovered – Interview with Dr. Gitler

Dr. Aaron Gitler and his colleagues recently published a paper in the August 12th issue of Science uncovering a potential new therapeutic target aimed at C9orf72 ALS, supported by The ALS Association. He found that inhibiting just one protein, called Spt4, significantly reduced toxicity caused by the C9orf72 repeat expansion. Learn more about how he used a simple model, yeast, to make this discovery and its therapeutic potential to treat ALS. And join us on November 7th to hear from Dr. Gitler during The ALS Association research webinar series (see details below).