Antisense oligonucleotide induced exon skipping and the dystrophin gene transcript: cocktails and chemistries.

ABSTRACT:

Background

Antisense oligonucleotides (AOs) can interfere with exon recognition and intron removal during pre-mRNA processing, and induce excision of a targeted exon from the mature gene transcript. AOs have been used in vitro and in vivo to redirect dystrophin pre-mRNA processing in human and animal cells. Targeted exon skipping of selected exons in the dystrophin gene transcript can remove nonsense or frame-shifting mutations that would otherwise have lead to Duchenne Muscular Dystrophy, the most common childhood form of muscle wasting.

Results

Although many dystrophin exons can be excised using a single AO, several exons require two motifs to be masked for efficient or specific exon skipping. Some AOs were inactive when applied individually, yet pronounced exon excision w

SUBMITTER: Adams AM 

PROVIDER: S-EPMC1933433 | biostudies-literature | 2007 Jul

REPOSITORIES: biostudies-literature