Josephus Johannes De Kimpe,Adriana Marie Rus,Gerard Johannes Platenburg,Judith Christina Theodora Van Deutekom,Garrit-Jan Boudewijn Van Ommen
申请号：
US16584115
公开号：
US20200239886A1
申请日:
2019.09.26
申请国别(地区):
US
年份:
2020
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 said cell with an antisense molecule that binds to a continuous stretch of at least 21 nucleotides within said exon. The invention further relates to such antisense molecule used in said method.
