Two-birds-one-stone strategy shows promise in RNA-repeat expansion diseases – Phys.org

A new strategy for treating a variety of diseases known as RNA-repeat expansion disorders, which affect millions of people, has shown promise in proof-of-principle tests conducted by scientists at Scripps Research.
The results suggest that someday, a handful of well-targeted drugs might be able to treat the more than 40 human disordersincluding Huntington’s disease and variants of amyotrophic lateral sclerosis (ALS)that arise from RNA-repeat expansions.
“This study lays a foundation for the development…

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