Aartsma-Rus, Annemieke,Van Ommen, Garrit-Jan Boudewijn,Van Deutekom, Judith Christina Theodora
申请号：
AU2006237727
公开号：
AU2006237727B2
申请日:
2006.04.21
申请国别(地区):
AU
年份:
2012
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 binding of an SR protein and/or 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.
