Scripps researchers create drug compound in effort to combat genetic disorders

Compound used to stop RNA sequence repeating that causes several diseases previously seen as incurable.


Photo of The Scripps Research Institute in Florida courtesy of Wikimedia Commons.

Ryan Lynch, Multimedia Editor


cientists working at The Scripps Research Institute on Florida Atlantic’s Jupiter campus have created a new drug that could fight several incurable genetic diseases caused by RNA defects.

The study saw researchers test their drug compound on the cells of patients with the genetic disorder Spinocerebellar Ataxia Type 10, or SCA10. After exposure to the compound, their cellular health improved.

According to TSRI professor Matthew Disney in an FAU news release, there are more than 30 incurable diseases caused by RNA repeats.

“By a thorough basic science investigation, we identified small molecules that target RNA base pairs precisely,” said Disney. “We then leveraged this information to design the first drug candidate that binds to disease-causing defects in SCA10. Application of the drug candidate returns certain aspects of those cells to healthy levels it’s like the defect is not even there.”

Disorders that are also caused by repeating RNA sequences include Huntington’s disease, fragile X-associated tremor ataxia syndrome and a form of muscular dystrophy.

Several potential drug candidates have been created using a database consisting of the best methods of interaction between RNA structures and drug compounds.

Disney said, “We are in the process of developing tools that allow one to design small molecules to target any RNA structural motif in a complex cellular environment.”

Ryan Lynch is the multimedia editor of the University Press. For information regarding this or other stories, email [email protected] or tweet him @RyanLynchwriter.