MÉTODOS E MEIOS PARA O SALTO EFICIENTE DO EXÃO 45 NO PRÉ-MRNA DE DISTROFIA MUSCULAR DE DUCHENNE

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专利权人:

BIOMARIN TECHNOLOGIES B.V.,;ACADEMISCH ZIEKENHUIS LEIDEN

发明人:

GARRIT-JAN BOUDEWIJN VAN OMMEN,GERARDUS JOHANNES PLATENBURG,JOSEPHUS JOHANNES DE KIMPE,JUDITH CHRISTINA THEODORA VAN DEUTEKOM,ANNEMIEKE AARTSMA-RUS

申请号：

PT13160338

公开号：

PT2607484E

申请日:

2009.01.13

申请国别(地区):

PT

年份:

2016

代理人:

摘要：

The invention relates to a method for inducing or promoting skipping of exon 45 of DMD pre-mRNA in a Duchenne Muscular Dystrophy patient, preferably in an isolated (muscle) cell, the method comprising providing said cell with a molecule that binds to a continuous stretch of at least 21 nucleotides within said exon.

来源网站：

中国工程科技知识中心

来源网址：

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