Duke Researchers Show "Editing" Can Fix Faulty Genes In New Approach To Ge...( plice in a corrected version. To accomp...)

plice in a corrected version. To accomplish this, he turned to the first ribozyme discovered, from the single-celled organism Tetrahymena thermophila. This ribozyme not only cuts other pieces of RNA at specific sites called recognition sequences, but after it cuts, it splices in a piece of RNA sequence attached to its tail end.

To test his idea of repairing faulty genes, Sullenger introduced a defective gene into mouse cells growing in a test tube. Then he engineered a ribozyme to recognize a short stretch of RNA near the genetic defect and splice in the corrected sequence, which Sullenger had produced artificially and attached to the ribozyme tail. When he introduced the ribozyme into the mouse cell, it recognized the defective RNA and swapped in the corrected version.

Although the technique is still in the proof-of-concept stage, Sullenger's twist on gene therapy addresses many of the problems that have complicated early gene therapy efforts.

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Contact: Karyn Hede George
georg016@mc.duke.edu
919-660-1301
Duke University
29-May-1996

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