Methods and means for efficient skipping of at least one of the following exons of the human Duchenne muscular dystrophy gene: 43, 46, 50- 53.

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专利权人:

Academisch Ziekenhuis Leiden;Prosensa Technologies B.V.

发明人:

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

申请号：

AU2009310558

公开号：

AU2009310558A8

申请日:

2009.03.11

申请国别(地区):

AU

年份:

2015

代理人:

摘要：

The invention relates a method wherein a molecule is used for inducing and/or promoting skipping of at least one of exon 43, exon 46, exons 50-53 of the DMD pre- mRNA in a patient, preferably in an isolated cell of a patient, the method comprising providing said cell and/or said patient with a molecule. The invention also relates to said molecule as such.

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