Methods and means for efficient skipping of exon 45 in Duchenne muscular dystrophy pre-MRNA

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专利权人:

PROSENSA HOLDING BV;ACADEMISCH ZIEKENHUIS LEIDEN;PROSENSA TECHNOLOGIES B.V.

发明人:

VAN DEUTEKOM, JUDITH CHRISTINA THEODORA,AARTSMA-RUS, ANNEMIEKE,VAN OMMEN, GARRIT-JAN BOUDEWIJN,PLATENBURG, GERARDUS JOHANNES,DE KIMPE, JOSEPHUS JOHANNES

申请号：

DK09788160

公开号：

DK2344637T3

申请日:

2009.01.13

申请国别(地区):

DK

年份:

2015

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 a molecule that binds to a continuous stretch of at least 21 nucleotides within said exon.

来源网站：

中国工程科技知识中心

来源网址：

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