MEANS AND METHOD FOR INDUCING EXON-SKIPPING

摘要

Provided is a method for determining whether the efficiency can be increased with which a dystrophin exon is excluded from a mature dystrophin mRNA in a cell that produces an exon containing precursor of said mRNA said method comprising providing said cell with a first anti-sense oligonucleotide (AON) that can hybridise to an exon internal part of said exon, and determining whether said efficiency is increased upon providing said cell with a second AON that can hybridise to another exon internal part of said exon, wherein said dystrophin exon is exon 45. The method is useful in regulating aberrant proteins in cells, including tumor cells.