BIOMARIN TECHNOLOGIES B;ANNEMIEKE AARTSMA-RUS;JOSEPHUS JOHANNES DE KIMPE;ACADEMISCH ZIEKENHUIS LEIDEN;GERARDUS JOHANNES PLATENBURG;JUDITH CHRISTINA THEODORA VAN DEUTEKOM;GARRIT-JAN BOUDEWIJN VAN OMMEN
发明人:
GERARDUS JOHANNES PLATENBURG,JUDITH CHRISTINA THEODORA VAN DEUTEKOM,ANNEMIEKE AARTSMA-RUS,JOSEPHUS JOHANNES DE KIMPE,GARRIT-JAN BOUDEWIJN VAN OMMEN
申请号：
IL20930110
公开号：
IL209301A
申请日:
2010.11.14
申请国别(地区):
IL
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.
