Synthesis and Exon-Skipping Properties of a 3'-Ursodeoxycholic Acid-Conjugated Oligonucleotide Targeting <i>DMD</i> Pre-mRNA: Pre-Synthetic versus Post-Synthetic Approach.

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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] ; Matteo,Bovolenta ^[2] ; Lorenzo,Preti ^[3] ; Massimo L,Capobianco ^[4] ; Kamel,Mamchaoui ^[5] ; Monica,Bertoldo ^[6] ; Daniela,Perrone ^[1]

作者单位： Department of Environmental and Prevention Sciences, University of Ferrara, 44121 Ferrara, Italy. ^[1] Department of Translational Medicine, University of Ferrara, 44121 Ferrara, Italy. ^[2] Department of Chemical, Pharmaceutical and Agricultural Sciences, University of Ferrara, 44121 Ferrara, Italy. ^[3] Institute of Organic Synthesis and Photoreactivity, Italian National Research Council, 40129 Bologna, Italy. ^[4] Centre de Recherche en Myologie, Institut de Myologie, Sorbonne Université, Inserm, F-75013 Paris, France. ^[5] Department of Chemical, Pharmaceutical and Agricultural Sciences, University of Ferrara, 44121 Ferrara, Italy.;Institute of Organic Synthesis and Photoreactivity, Italian National Research Council, 40129 Bologna, Italy. ^[6]

关键词 antisense oligonucleotide bile acid conjugation dystrophin exon-skipping solid phase synthesis solid support ursodeoxycholic acid