Sposób skutecznego pominięcia eksonu (44) w dystrofii mięśniowej Duchenne'a i związane z nim środki

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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：

PL14179979

公开号：

PL2813570T3

申请日:

2009.05.14

申请国别(地区):

PL

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.

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