Exon exchange approach to repair Duchenne dystrophin transcripts.

ABSTRACT:

Background

Trans-splicing strategies for mRNA repair involve engineered transcripts designed to anneal target mRNAs in order to interfere with their natural splicing, giving rise to mRNA chimeras where endogenous mutated exons have been replaced by exogenous replacement sequences. A number of trans-splicing molecules have already been proposed for replacing either the 5' or the 3' part of transcripts to be repaired. Here, we show the feasibility of RNA surgery by using a double trans-splicing approach allowing the specific substitution of a given mutated exon.

Methodology/principal findings

As a target we used a minigene encoding a fragment of the mdx dystrophin gene enclosing the mutated exon (exon 23). This minigene was cotransfected with a variety of exon exchange constru

SUBMITTER: Lorain S 

PROVIDER: S-EPMC2878348 | biostudies-literature | 2010 May

REPOSITORIES: biostudies-literature