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Antisense oligonucleotide directed removal of proteolytic cleavage sites from proteins
专利权人:
ACADEMISCH ZIEKENHUIS LEIDEN H.O.D.N. LUMC
发明人:
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.
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