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Jul 16, 2020

RNA repair shows promise in reversing mutations underlying a neurological disorder

Posted by in categories: biotech/medical, genetics

Scientists successfully edited RNA in a living animal in such a way that the repaired RNA then corrected a mutation in a protein that gives rise to a debilitating neurological disorder in people known as Rett syndrome.

The advance by researchers at Oregon Health & Science University publishes in the journal Cell Reports.

“This is the first example of using programmable RNA editing to repair a gene in mouse models of a neurological disease,” said senior author Gail Mandel, Ph.D., senior scientist in the OHSU Vollum Institute. “This gives us an approach that has some traction.”

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