Methods and means for efficient skipping of exon 45 in Duchenne Muscular Dystrophy pre-mRNA

• 收藏
• 分享

专利权人:

Academisch Ziekenhuis Leiden;Prosensa Holding BV;Prosensa B.V.;Prosensa Technologies B.V.

发明人:

De Kimpe, Josephus Johannes,Platenburg, Gerardus Johannes,Van Deutekom, Judith Christina Theodora,Aartsma-Rus, Annemieke,Van Ommen, Garrit-Jan Boudewijn

申请号：

AU2009310557

公开号：

AU2009310557B2

申请日:

2009.01.13

申请国别(地区):

AU

年份:

2014

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.

来源网站：

中国工程科技知识中心

来源网址：

http://www.ckcest.cn/home/