Antisense Oligonucleotides Conjugated with Lipophilic Compounds: Synthesis and In Vitro Evaluation of Exon Skipping in Duchenne Muscular Dystrophy.

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第一作者： Elena,Marchesi

第一单位： Department of Environmental and Prevention Sciences, University of Ferrara, 44121 Ferrara, Italy.

作者： Elena,Marchesi ^[1] ; Rita,Cortesi ^[2] ; Lorenzo,Preti ^[2] ; Paola,Rimessi ^[3] ; Maddalena,Sguizzato ^[2] ; Matteo,Bovolenta ^[4] ; Daniela,Perrone ^[1]

作者单位： Department of Environmental and Prevention Sciences, University of Ferrara, 44121 Ferrara, Italy. ^[1] Department of Chemical, Pharmaceutical and Agricultural Sciences, University of Ferrara, 44121 Ferrara, Italy. ^[2] Unit of Medical Genetics, S. Anna University-Hospital, 44121 Ferrara, Italy. ^[3] Department of Translational Medicine, University of Ferrara, 44121 Ferrara, Italy. ^[4]

关键词 2′-O-methyl-phosphorothioate (2′-OMe PS)Duchenne muscular dystrophy antisense oligonucleotide bile acid conjugation exon skipping lipophilic compound ursodeoxycholic acid (UDCA)

主题词细胞系(Cell Line);肌营养不良蛋白(Dystrophin);外显子(Exons);人类(Humans);肌营养不良, 杜氏(Muscular Dystrophy, Duchenne);寡核苷酸类(Oligonucleotides);寡核苷酸类, 反义(Oligonucleotides, Antisense)

DOI 10.3390/ijms23084270

PMID 35457088

发布时间 2022-07-16

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