Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means

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专利权人:

BioMarin Technologies B.V.;Academisch Ziekenhuis Leiden

发明人:

PLATENBURG, Gerardus Johannes,DE KIMPE, Josephus Johannes,VAN DEUTEKOM, Judith Christina Theodora,VAN OMMEN, Garrit-Jan Boudewijn

申请号：

AU2019283969

公开号：

AU2019283969A1

申请日:

2019.12.20

申请国别(地区):

AU

年份:

2020

代理人:

摘要：

#$%^&*AU2019283969A120200123.pdf#####Abstract The invention relates to a nucleic acid molecule that binds and/or is complementary to the nucleotide molecule having sequence 5'GUGGCUAACAGAAGCU (SEQ ID NO 1) and to its use in a method for inducing 5 skipping of exon 44 of the DMD gene in a DMD patient. 11982666_1 (GHMatters) P85642.AU.3

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