Johannes Theodorus den Dunnen;Annemieke Aartsma-Rus;Garrit-Jan Boudewijn van Ommen;Judith Christina Theodora van Deutekom
发明人:
Garrit-Jan Boudewijn van Ommen,Judith Christina Theodora van Deutekom,Johannes Theodorus den Dunnen,Annemieke Aartsma-Rus
申请号：
US11233495
公开号：
US09896687B2
申请日:
2005.09.21
申请国别(地区):
US
年份:
2018
代理人:
摘要：
The invention provides a method for generating an oligonucleotide with which an exon may be skipped in a pre-mRNA and thus excluded from a produced mRNA thereof. Further provided are methods for altering the secondary structure of an mRNA to interfere with splicing processes and uses of the oligonucleotides and methods in the treatment of disease. Further provided are pharmaceutical compositions and methods and means for inducing skipping of several exons in a pre-mRNA.
