VAN DEUTEKOM JUDITH CHRISTINA THEODORA,DE KIMPE JOSEPHUS JOHANNES,PLATENBURG GERARDUS JOHANNES
申请号：
IN8036/CHENP/2011
公开号：
IN2011CN08036A
申请日:
2011.11.02
申请国别(地区):
IN
年份:
2013
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.
