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Although only 10 percent of amyotrophic lateral sclerosis (ALS) cases are hereditary, a significant number of them are caused by mutations that affect proteins that bind RNA, a type of genetic material. University of California, San Diego, School of Medicine researchers studied several ALS cases with a mutation in an RNA-binding protein known as hnRNP A2/B1.
In the study, they describe how damage to this protein contributes to ALS by scrambling crucial cellular messaging systems. The research team says these findings are a significant step forward in validating RNA-based therapy as a treatment for ALS.Image credit: UC San Diego Health
