Targeting Disease-Causing RNAs in Precision Medicine
February 14, 2017 | Terry Sharrer
“With the precision of a surgeon’s scalpel, we have shown that small molecules can be designed to seek out and destroy only disease-causing RNAs. Further, we developed novel chemical approaches to use a disease-causing RNA to help make its own drug by using that RNA as a catalyst for drug synthesis at the needed site. It is like having your physician place a drug at the right place without exposing healthy cells.” This is how biochemists at The Scripps Research Institute (Jupiter, FL) describe their highly innovative approach for treating myotonic dystrophy type 1, with implications for other inherited RNA diseases. MORE
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