METHOD FOR EFFICIENT EXON (44) SKIPPING IN DUCHENNE MUSCULAR DYSTROPHY AND ASSOCIATED MEANS

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专利权人:

ACADEMISCH ZIEKENHUIS LEIDEN;PROSENSA TECHNOLOGIES B.V.

发明人:

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

申请号：

NZ60018309

公开号：

NZ600183A

申请日:

2009.05.14

申请国别(地区):

NZ

年份:

2013

代理人:

摘要：

600183 Provided is a nucleic acid molecule that binds and/or is complementary to nucleotide sequence: 5'-CUGUUGAGAAA whereby said molecule comprises 23, 24, 25, 26 or 27 nucleotides and comprises or consists of the base sequence of GCCAUUUCUCAACAGAUCUGUCA, wherein optionally one or more of said nucleotides is a nucleotide analogue. The

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