WILHELMUS WENCESLAUS MATTHIAS PIJNAPPEL,ANTJE TJITSKE VAN DER PLOEG,ERIK VAN DER WAL,ATZE JACOBUS BERGSMA
申请号：
NL2017294
公开号：
NL2017294B1
申请日:
2016.08.05
申请国别(地区):
NL
年份:
2018
代理人:
摘要：
The invention relates to method for repairing aberrant splicing, wherein such aberrant splicing is caused by the expression of a natural pseudo exon, comprising blocking of either the natural cryptic 3’ splice site or the natural cryptic 5’ splice site of said natural pseudo exon with an antisense oligomeric compound (AON). Further, the invention comprises an antisense oligomeric compound targeting SEQ ID NO:1 or SEQ ID NO: 180, preferably selected from the sequences of SEQ ID NO: 91 – 179, sequences that are complementary to said sequences or sequences that have an identity of 80% with said sequences or the complementary sequences and a second AON from the sequences of SEQ ID NO: 346 – 508, sequences that are complementary to said sequences or sequences that have an identity of 80% with said sequences or the complementary sequences. These AONs are specifically for use in the treatment of Pompe disease.
