Procédé pour l'omission efficace de l'exon (44) dans la dystrophie musculaire de Duchenne et moyens connexes

• 收藏
• 分享

专利权人:

BioMarin Technologies B.V.;Academisch Ziekenhuis Leiden

发明人:

申请号：

EP14179979.1

公开号：

EP2813570B1

申请日:

2009.05.14

申请国别(地区):

EP

年份:

2017

代理人:

摘要：

The invention provides a method for modulating skipping of Duchenne Muscular Dystrophy (DMD) gene in a cell. More specifically, the invention provides methods and means for modulating skipping of exon 44 of DMD. The invention further provides nucleic acid molecules that can be used for a method of the invention, expression methods for expression of a nucleic acid molecule in a cell, and use of a nucleic acid molecule for modulating skipping of DMD in a cell.

来源网站：

中国工程科技知识中心

来源网址：

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