Josephus Johannes De Kimpe;Gerardus Johannes Platenburg;Judith Christina Theodora Van Deutekom
发明人:
Judith Christina Theodora Van Deutekom,Josephus Johannes De Kimpe,Gerardus Johannes Platenburg
申请号：
US13266110
公开号：
US20120046342A1
申请日:
2010.04.26
申请国别(地区):
US
年份:
2012
代理人:
摘要：
The invention provides an oligonucleotide comprising an inosine, and/or a nucleotide containing a base able to form a wobble base pair or a functional equivalent thereof, wherein the oligonucleotide, or a functional equivalent thereof, comprises a sequence which is complementary to at least part of a dystrophin pre-m RNA exon or at least part of a non-exon region of a dystrophin pre-m RNA said part being a contiguous stretch comprising at least 8 nucleotides. The invention further provides the use of said oligonucleotide for preventing or treating DMD or BMD.
