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Scientists uncover image of muscular dystrophy defect & design targeted drug candidates
Date:1/2/2014

The Scripps Florida team spent several years on the problem and succeeded in engineering the RNA to have crystal contacts in different positions. This allowed the RNA to be crystallizedand its structure to be revealed.

Using information about the RNA's structure and movement, the scientists were able to design molecules to improve RNA function.

The new findings were confirmed using sophisticated computational models that show precisely how the small molecules interact with and alter the RNA structure over time. Those predictive models matched what the scientists found in the studythat these new compounds bind to the repeat structure in a predictable and easily reproducible way, attacking the cause of the disease.

"We used a bottom-up approach, by first understanding how the small components of the RNA structure interact with small molecules," said Jessica Childs-Disney of TSRI, who was first author of the paper with Ilyas Yildirim of Northwestern University. "The fact that our compounds improve the defects shows that our unconventional approach works."


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Contact: Eric Sauter
esauter@scripps.edu
267-337-3859
Scripps Research Institute
Source:Eurekalert  

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Scientists uncover image of muscular dystrophy defect & design targeted drug candidates
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