VAN ROON-MOM WILHELMINA MARIA CLASINA,EVERS MELVIN MAURICE,PEPERS BARRY ANTONIUS,AARTSMA-RUS ANNEMIEKE,VAN OMMEN GARRIT-JAN BOUDEWIJN
申请号：
NZ60729711
公开号：
NZ607297A
申请日:
2011.08.04
申请国别(地区):
NZ
年份:
2015
代理人:
摘要：
Discloses a method for removing a proteolytic cleavage site from a protein comprising providing a cell that expresses pre-mRNA encoding said protein ex vivo with an anti-sense oligonucleotide that induces skipping of the exonic sequence that encodes said proteolytic cleavage site, the method further comprising allowing translation of mRNA produced from said pre-mRNA. Further discloses an oligonucleotide of between 14-40 nucleotides that induces skipping of an exon that encodes a proteolytic cleavage site in a protein, wherein said protein is involved in a neurodegenerative disorder.
