Pathogenic RNA repeats contribute to disorders including Huntington's disease, fragile X-associated tremor ataxia syndrome and myotonic dystrophy type 1 and 2.
Scientists for the first time created a drug candidate that attacks and neutralizes the RNA structure that causes an incurable progressive, inherited disease involving a gradual loss of control over body movement.
The study, which was published in Nature Communications, showed the compound significantly improved several aspects of cells taken directly from patients with spinocerebellar ataxia type 10 (SCA10), a form of spinocerebellar ataxia.
"More than 30 diseases, all of them incurable, are caused by RNA repeats," said study lead. "By a thorough basic science investigation, we identified small molecules that target RNA base pairs precisely. We then leveraged this information to design the first drug candidate that binds to disease-causing defects in SCA10. Application of the drug candidate returns certain aspects of those cells to healthy levels--it's like the defect is not even there."
SCA10 is caused by what is called a pentanucleotide repeat (a genetic sequence of five nucleotides repeated many more times than normal) affecting the mitochondria, the cell's energy source. The new drug candidate, known as 2AU-2, targets these repeats by binding to RNA base pairs.
"The potent bioactivity of 2AU-2 to moderate the structurally induced toxicity in SCA10 strongly suggests that base-pair-targeting RNA modules could have broad applicability in our effort to develop other compounds that target different RNAs," said the first author of the study. "More than 70 percent of RNA secondary structure is made up of base pairing."
http://www.scripps.edu/news/press/2016/20160601disney.html
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