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Hepatocyte‐Specific Delivery of siRNAs Conjugated to Novel Non‐nucleosidic Trivalent N‐Acetylgalactosamine Elicits Robust Gene Silencing in Vivo
ChemBioChem ( IF 2.6 ) Pub Date : 2015-03-18 , DOI: 10.1002/cbic.201500023
Kallanthottathil G. Rajeev , Jayaprakash K. Nair , Muthusamy Jayaraman , Klaus Charisse , Nate Taneja , Jonathan O'Shea , Jennifer L. S. Willoughby , Kristina Yucius , Tuyen Nguyen , Svetlana Shulga‐Morskaya , Stuart Milstein , Abigail Liebow , William Querbes , Anna Borodovsky , Kevin Fitzgerald , Martin A. Maier , Muthiah Manoharan

Sugar goes straight to the liver: An siRNA conjugate bearing three N‐acetylgalactosamine moieties, each sequentially attached by a non‐nucleosidic linker, was recognized by the asialoglycoprotein receptor with high affinity. The resulting liver‐specific delivery of the conjugate for robust RNAi‐mediated gene silencing in vivo shows potential for delivery of oligonucleotide therapeutics into hepatocytes.
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中文翻译:

与新型非核苷三价N-乙酰半乳糖胺偶联的特异性针对肝细胞的siRNA导致体内的稳健基因沉默

糖直接进入肝脏:带有3个N-乙酰半乳糖胺部分的siRNA偶联物,每个由一个非核苷接头依次连接,被去唾液酸糖蛋白受体识别为高亲和力。结合物在肝脏中的特异性递送,可在体内实现强大的RNAi介导的基因沉默,显示出将寡核苷酸治疗剂递送至肝细胞的潜力。
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更新日期:2015-03-18
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