DIRECTED BY ANTI-SENSE OLIGONUCLEOTID, DELETING PROTEOLYTIC SPLITTING WEBSITES, HCHWA-D MUTATION AND AN INCREASED NUMBER OF TRINUCLEOTIDE REPEATS

摘要

The invention relates to means and methods for removing a proteolytic cleavage site, the HCHWA-D mutation or the amino acids encoded by a trinucleotide repeat expansion from a protein comprising providing a cell that expresses pre-m RNA encoding said protein with an anti-sense oligonucleotide that induces skipping of the exonic sequence that comprises said proteolytic cleavage site, HCHWA-D mutation or trinucleotide repeat expansion, respectively, the method further comprising allowing translation of m RNA produced from said pre-m RNA.