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Antisense compounds inducing exon skipping and compositions comprising same for treating muscular dystrophy
Antisense compounds inducing exon skipping and compositions comprising same for treating muscular dystrophy
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机译：诱导外显子跳跃的反义化合物和包含其的组合物用于治疗肌营养不良症
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摘要
The present invention relates to an oligonucleotide which specifically hybridizes to a target region in an exon of the human dystrophin gene inducing exon skipping. The present invention further relates to a pharmaceutical composition comprising an oligonucleotide which specifically hybridizes to a target region in an exon of the human dystrophin gene inducing exon skipping.
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