May 1, 1998 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, profe