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ANTISENSE POLYNUCLEOTIDES TO INDUCE EXON SKIPPING AND METHODS OF TREATING DYSTROPHIES
ANTISENSE POLYNUCLEOTIDES TO INDUCE EXON SKIPPING AND METHODS OF TREATING DYSTROPHIES
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机译:诱导外显子跳跃的反义多核苷酸和治疗营养不良的方法
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摘要
Antisense polynucleotides and their use in pharmaceutical compositions to induce exon skipping in targeted exons of the gamma sarcoglycan gene are provided, along with methods of preventing or treating dystrophic diseases such as Limb-Girdle Muscular Dystrophy. One aspect of the disclosure provides an isolated antisense polynucleotide wherein the polynucleotide specifically hybridizes to an exon target region of a gamma sarcoglycan RNA, wherein the exon is selected from the group consisting of exon 4 (SEQ ID NO: 1), exon 5 (SEQ ID NO: 2), exon 6 (SEQ ID NO: 3), exon 7 (SEQ ID NO: 4) and a combination thereof. In some embodiments, the antisense polynucleotide cannot form an RNase H substrate, and in further embodiments the antisense polynucleotide comprises a modified polynucleotide backbone.
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机译:提供了反义多核苷酸及其在药物组合物中的诱导γ肌糖蛋白基因的靶向外显子中外显子跳跃的用途,以及预防或治疗营养不良性疾病例如肢带型肌营养不良症的方法。本公开内容的一个方面提供了一种分离的反义多核苷酸,其中该多核苷酸与γ-肌聚糖RNA的外显子靶区域特异性杂交,其中该外显子选自外显子4(SEQ ID NO:1),外显子5(SEQ ID NO:1)。 (ID NO:2),外显子6(SEQ ID NO:3),外显子7(SEQ ID NO:4)及其组合。在一些实施方案中,反义多核苷酸不能形成RNase H底物,并且在进一步的实施方案中,反义多核苷酸包含修饰的多核苷酸主链。
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