Novel RNA-modifying tool corrects genetic diseases
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IMAGE: Professor Matthew Disney of The Scripps Research Institute led the new study. view more 

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Credit: The Scripps Research Institute

JUPITER, FL–May 29, 2018–As scientists gain insights into which genes drive diseases, they are pursuing the next logical question: Can gene editing technologies be developed to treat or even cure those diseases? Much of that effort has focused on developing technologies such as CRISPR-Cas9, a protein-based system.

At The Scripps Research Institute campus in Florida, chemist Matthew D. Disney, PhD, has taken a different approach, developing a small-molecule-based tool that acts on RNA to selectively delete certain gene products.

Disney’s deletion tool opens the possibility of creating drugs that can be taken conveniently as pills to correct genetic diseases–by destroying toxic gene products, and by chemically controlling the body’s defense mechanisms. The paper, “Small molecule targeted recruitment of a nuclease to RNA,” was published online by the Journal of the American Chemical Society.

“These studies, like much science, were about a decade in the making. We are very excited to see how this initial application evolves,” Disney says. “This research further shows that RNA is indeed a viable target to make medicines.”

RNAs represent a diverse group of molecules within cells that act like the cells’

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