Methods and means for efficient skipping of at least one of the exons 51-53, 55, 57 and 59 of the human duchenne muscular dystrophy gene

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专利权人:

Academisch Ziekenhuis Leiden;Prosena Technologies B.V.

发明人:

Judith Christina Theodora van Deutekom

申请号：

US14631686

公开号：

US09499818B2

申请日:

2015.02.25

申请国别(地区):

US

年份:

2016

代理人:

摘要：

The invention relates to a method where a molecule is used for inducing and/or promoting the skipping of at least one of the exons 51-53, 55, 57 and 59 of the DMD pre-mRNA in a patient, preferably in an isolated cell of a patient. The method comprising providing the cell and/or the patient with a molecule. The invention also relates to the molecule and its composition that is being used for inducing exon skipping.

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中国工程科技知识中心

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