Article
A reporter-based screen to identify potent 3' trans-splicing molecules for endogenous RNA repair.
Human gene therapy methods - 1 Feb 2013
Murauer Eva M, Koller Ulrich, Hainzl Stefan, Wally Verena, Bauer Johann W
Abstract excerpt
In the treatment of genetic disorders, repairing defective pre-mRNAs by RNA trans-splicing has become an emerging alternative to conventional gene therapy. Previous studies have made clear that the design of the binding domains of the corrective RNA trans-splicing molecules (RTMs) is crucial for their optimal functionality. We established a reporter-based screening method that allows for selection of highly...
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