Article
Using 5'-PTMs to repair mutant beta-globin transcripts.
RNA (New York, N.Y.) - 1 Aug 2007
Kierlin-Duncan Monique N, Sullenger Bruce A
Abstract excerpt
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...
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