RNA modulation, repair and remodeling by splice switching oligonucleotides

"Targeting splicing by antisense oligonucleotides allows RNA modifications that
are not possible with RNA interference or other antisense techniques that destine
the RNA for destruction. By changing the ratio of naturally occurring splice variants
the expression of mRNA is modulated. By preventing the use of an aberrant splice
site created by a mutation and enforcing re-selection of correct splice sites the RNA
is repaired. Antisense induced skipping of the exon that carries a nonsense mutation
remodels the mRNA and restores the reading frame of the defective protein. All of
the above approaches have clinical applications. Modulation of splice variants is particularly
important since close to 60% of all genes code for alternatively spliced pre-mRNA."

Ryszard Kole, Tiffany Williams and Lisa Cohen
RNA modulation, repair and remodeling by splice switching oligonucleotides
Acta Biochimica Polonica Vol. 51 No. 2/2004