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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