Researchers find drug that may suppress genetic mutation using a novel screening approach
Researchers at Massachusetts General Hospital have developed a way of identifying promising new drugs that may get around a major challenge in drug discovery. In the May issue of Nature Biotechnology the team from the MGH Cardiovascular Research Center (CVRC) describes using an animal model to screen for a compound that suppresses a serious genetic mutation. Their success did not rely on first identifying a molecular target for the new drug, something that is a key bottleneck in current procedures. The study is being released today on the journal’s website.
"Currently most drugs are designed to act on a specific protein, but for most diseases we still don’t know what the protein targets should be," says Randall Peterson, PhD, of the MGH CVRC, the paper’s lead author. "This is a totally different approach that shows how, without knowing the best target, you can screen for drugs that could reverse a disease and in the process learn something new about the underlying biology."
Sue McGreevey | EurekAlert!
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