Fremgangsmåder og midler til effektiv overspringning af exon 45 i Duchennes muskeldystrofi pre-MRNA

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专利权人:

BioMarin Technologies B.V.;Academisch Ziekenhuis Leiden

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：

DK20090788160T

公开号：

DK2344637(T4)

申请日:

2009.01.13

申请国别(地区):

丹麦

年份:

2018

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/