Antisense Oligonucleotides Conjugated with Lipophilic Compounds: Synthesis and in Vitro Evaluation of Exon Skipping in Duchenne Muscular Dystrophy
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES（2022）
关键词
antisense oligonucleotide,exon skipping,conjugation,2 '-O-methyl-phosphorothioate (2 '-OMe PS),bile acid,ursodeoxycholic acid (UDCA),lipophilic compound,Duchenne muscular dystrophy
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