POSTUPAK DJELOTVORNOG PRESKAKANJA EGZONA (44) KOD DUCHENNEOVE MIŠIĆNE DISTROFIJE I ODGOVARAJUĆE SREDSTVO

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专利权人:

BioMarin Technologies B.V.;Academisch Ziekenhuis Leiden

发明人:

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

申请号：

HRP20171980

公开号：

HRP20171980T1

申请日:

2017.12.20

申请国别(地区):

HR

年份:

2018

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

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