Professor Matthew Disney of The Scripps Research Institute led the new study. Credit: The Scripps Research Institute
A new tool opens the possibility of creating drugs that can be taken conveniently as pills to correct genetic diseases. 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...
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