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

机译:高效外显子(44)跨越Duchenne肌营养不良和相关手段的方法

摘要

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), 5′-GGGAACAUGCUAAAUAC (SEQ ID NO:2), 5′-AGACACAAAUUCCUGAGA (SEQ ID NO:3), or 5′-CUGUUGAGAAA (SEQ ID NO. 4), and to its use in a method for inducing skipping of exon 44 of the DMD gene in a DMD patient.
机译:本发明涉及结合和/或与具有序列5'-guggcuaacagaagcu(SEQ ID NO:1),5'-gggaaCaugcuaauac(SEQ ID NO:2),5'-agacacaauccugaga()的核苷酸分子互补的核酸分子SEQ ID NO:3),或5'-cuguugagaAA(SEQ ID NO.4),并在DMD患者中诱导DMD基因的外显子44的方法。

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