Josephus Johannes De Kimpe,Adriana Marie Rus,Gerard Johannes Platenburg,Judith Christina Theodora Van Deutekom,Garrit-Jan Boudewijn Van Ommen
申请号：
US14542183
公开号：
US09528109B2
申请日:
2014.11.14
申请国别(地区):
US
年份:
2016
代理人:
摘要：
The invention relates to a method for inducing or promoting skipping of exon 45 of DMD pre-mRNA in a Duchenne Muscular Dystrophy patient, preferably in an isolated (muscle) cell, the method comprising providing an isolated muscle cell with a molecule that binds to a continuous stretch of at least 21 nucleotides within the exon. The invention further relates to such molecule used in the method.
