METHODS AND MEANS FOR EFFICIENT SKIPPING OF EXON 52 OF THE HUMAN DUCHENNE MUSCULAR DYSTROPHY GENE

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专利权人:

ACADEMISCH ZIEKENHUIS LEIDEN;PROSENSA HOLDING BV;PROSENSA B.V.;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：

NZ59249809

公开号：

NZ592498A

申请日:

2009.03.11

申请国别(地区):

NZ

年份:

2012

代理人:

摘要：

Disclosed is a molecule comprising or consisting of the antisense nucleotide sequence: gguaaugaguucuuccaacugg. Said molecule binds to a continuous stretch of at least 8 nucleotides within the following nucleotide sequence: 5'AUGCAGGAUUUGGAACAGAGGCGUCCCCAGUUGGAAGAACUCAUUACCGCUGCCCAAAAUUUGAAAAACAAGACCAGCAAUCAAGAGGCU-3' for skipping of exon 52. The disclosed molecule is used in the treatment of Duchenne muscular dystrophy and Becker muscular dystrophy.

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