ALS and dementia attacked by an RNA-hunting compound that recruits cell’s own virus fighter

ALS and dementia attacked by an RNA-hunting compound that recruits cell's own virus fighter
To study the effectiveness of their compounds, the scientists directed cells from patients with ALS to become stem cells and then specialize into nerve cells. Credit: Jessica Bush, Disney Lab at Scripps Research, Florida

One of the most commonly inherited forms of ALS and frontotemporal dementia is referred to as C9 ALS/FTD, so named for the repeated section of DNA on chromosome 9 that causes it. A collaboration led by scientists at Scripps Research in Florida has successfully treated the genetic disease in mice, with a potential drug molecule engineered in the lab of chemist Matthew Disney, Ph.D. The compound works in a new way, by directing the cell’s own immune machinery to degrade and eliminate the disease-causing RNA.

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