Scientists at Duke University release new data on RNA research
2007 OCT 26 -- According to a study from the United States, "'Trans-splicing has been used to repair mutant RNA transcripts via competition for the spliceosome using pre-trans-splicing molecules, or ''PTMs.'' Previous studies have demonstrated that functional PTMs can be designed for either 3'- or 5'-exon replacement, with a vast majority of the work to date focusing on repair of mutations within internal exons and via 3'-exon replacement. Here, we describe the first use of trans- splicing to target the first exon and intron of a therapeutically relevant gene and repair the mutant RNA by 5'-exon replacement." "Our results show that 5'-PTMs can be designed to repair mutations in the beta-globin transcript involved in sickle cell anemia and b- thalassemia while providing insight into considerations for competition between trans- versus cis- splicing in mammalian cells. Target transcripts with impaired cis-splicing capabilities, like those produced in some forms of b-thalassemia, are more efficiently repaired via trans-splicing than targets in which cis- splicing is unaffected as with sickle b-globin," wrote M.N. Kierlinduncan and colleagues, Duke University. The researchers concluded: "This study reveals desirable characteristics in substrate RNAs for trans-splicing therapeutics as well as provides an opportunity for further exploration into general splicing mechanisms via 5'-PTMs." Kierlinduncan and colleagues published the results of their research in RNA - a Publication of the RNA Society (Using 59-PTMs to repair mutant beta-globin transcripts. RNA - a Publication of the RNA Society, 2007;13(8):1317-1327). For additional information, contact B.A. Sullenger, Duke University, Medical Center, Dept. of Surgery, Durham, NC 27708, USA. The publisher of the journal RNA - a Publication of the RNA Society can be contacted at: Cold Spring Harbor Laboratory Press, Publications Dept., 500 Sunnyside Blvd., Woodbury, NY 11797-2924, USA. Keywords: United States, Durham, Life Sciences, Interferon Alfa-2b, Intron, Pharmaceuticals, Drugs, Therapy, Treatment, Rna-a PublicationRna Society, Duke University. This article was prepared by Surgery Litigation & Law Week editors from staff and other reports. Copyright 2007, Surgery Litigation & Law Week via NewsRx.com.
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