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Antisense Compounds Targeting Genes Associated with Cystic Fibrosis
Antisense Compounds Targeting Genes Associated with Cystic Fibrosis
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机译:反义化合物靶向与囊性纤维化相关的基因
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摘要
The present disclosure relates generally to compounds comprising oligonucleotides complementary to a cystic fibrosis transmembrane conductance regulator (CFTR) RNA transcript. Certain such compounds are useful for hybridizing to a CFTR RNA transcript, including but not limited to a CFTR RNA transcript in a cell. In certain embodiments, such hybridization results in modulation of splicing and/or expression of the CFTR transcript. In certain embodiments, such compounds are used to treat one or more symptoms associated with Cystic Fibrosis.
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