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Pre-mRNA splice switching or regulatory oligonucleotides containing bicyclic scaffold moieties with improved characteristics for the treatment of genetic disorders
Pre-mRNA splice switching or regulatory oligonucleotides containing bicyclic scaffold moieties with improved characteristics for the treatment of genetic disorders
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机译:包含双环支架部分的前mRNA剪接转换或调控寡核苷酸,具有改善的特性,可用于治疗遗传性疾病
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摘要
The present invention provides antisense splice switching oligonucleotides with improved features that enhance clinical applicability to treat, ameliorate, prevent and / or delay neuromuscular disorders such as DMD. [Selection figure] None
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