Method for Effective Exon (44) Skipping in Duchenne Muscular Dystrophy and Similar Agents

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专利权人:

BioMarin Technologies B.V.;Academisch Ziekenhuis Leiden

发明人:

Platenburg, Gerard Johannes,DE KIMPE, Josephus Johannes,van Deutekom, Judith Christina Theodora,van Ommen, Garrit-Jan Boudewijn,AARTSMA-RUS, Annemieke

申请号：

DK14179979

公开号：

DK2813570T3

申请日:

2009.05.14

申请国别(地区):

DK

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.

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