New RNA Repair May Lead To More Successful Gene Therapy

CHAPEL HILL - A possible new form of gene therapy designed to mask genetic mutations - instead of cutting away and replacing them -- has been developed by scientists at the University of North Carolina at Chapel Hill School of Medicine and Bern University in Switzerland.

The technique, so far limited to laboratory cell cultures, involves using small RNA molecules to block defective processing, or splicing, inside cell nuclei of a messenger RNA that codes for a blood protein known as beta-globin.

Since the short RNA fragments block the faulty processing sites, cells' splicing machinery can only use functional, non-mutated locations, researchers say. What results is steady production of healthy "messengers," which then relay accurate genetic instructions into cell cytoplasm where normal proteins assemble.

A report on the research appears in the April 28 issue of the Proceedings of the National Academy of Sciences. Authors include pharmacology doctoral student Linda Gorman and Dr. Ryszard Kole, professor of pharmacology at UNC-CH.

"This work offers real hope that one day we will be able to cure - not just treat -- beta thalassemia, an inherited deficiency of hemoglobin, the essential protein that carries oxygen and gives blood its red color," said Kole, a member of the UNC Lineberger Comprehensive Cancer Center. "We are still a few steps away from trying this in patients, but it is quite promising."

Like sending imperfect plans to a factory, errors in messenger RNA production result in defective or inadequate protein production, he said. In severe cases of untreated beta thalassemia, those errors lead to acute anemia and death at a young age.

"The advantage of our novel way to block the incorrect splice sites is that the chances of doing something inappropriate to genes are minimal," Kole said. "A limitation would be that you can't

Contact: David L. Williamson
University of North Carolina at Chapel Hill

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