Induction of exon skipping of the dystrophin transcript in lymphoblastoid cells by transfecting an antisense oligodeoxynucleotide complementary to an exon recognition sequence.

In this paper we first report that exon skipping from the dystrophin gene transcript could be induced in living cells by an antisense oligodeoxynucleotide (ODN) complementary to an exon recognition sequence (ERS). Incubation of lymphoblastoid cells with an antisense ODN against the purine-rich region of dystrophin exon 19 resulted in skipping of the exon from the dystrophin transcript. Skipping of exon 19 started to appear after 6 hours of incubation, and complete skipping was observed after 24 hours of incubation. None of the other 78 dystrophin exons were skipped, and exon 19 skipping could be induced by the sense ODN or by an antisense ODN corresponding to another ERS. These results showed that antisense ODN against ERS can induce exon skipping even in living cells and ERS is functioning as an essential cis-element for proper splicing in dystrophin pre-mRNA.