Splicing ribozyme can 'edit' mammalian RNA

Abstract

In a novel approach to gene therapy, researchers at Duke University Medical Center have shown that ribozymes can edit genetic information encoded in messenger RNA molecules inside mammalian cells. If further development is successful, the technique could, in effect, bypass a defective gene and instead correct the RNA blueprint the gene produces to direct protein synthesis. Ribozymes are RNA segments that catalyze reactions within cells. Because they selectively bind and react with other RNA segments, they have drawn attention as potential tools that could protect against viral infection by cleaving and destroying an invading virus's RNA. Clinical trials are now testing the potential of several modified ribozymes to protect against HIV infection. But the Duke team's approach is quite different. Assistant professor of surgery and genetics Bruce A. Sullenger, research assistant Joshua T. Jones, and postdoctoral fellow Seong-Wook Lee modified a ribozyme from a different class than those that cleave viral RN...