Fletcher S, Adams AM, Johnsen RD, Greer K, Moulton HM, Wilton SD. Dystrophin Isoform Induction In Vivo by Antisense-mediated Alternative Splicing. Mol Ther. 2010 Mar 23. [Epub ahead of print] [Only registered users see links. ]
No animal model for a mutation? Wilton et al. use one Morpholino to alter splicing and phenocopy a mutation at the RNA level, then another Morpholino to remove another exon and restore the reading frame. The resulting protein has the same structure as one produced by a mutant lacking an exon that is treated with a frame-correcting oligo.