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- W3157717776 abstract "Small interfering RNA (siRNA) is a clinically approved therapeutic modality, which has attracted widespread attention not only from basic research but also from pharmaceutical industry. As siRNA can theoretically modulate any disease-related gene’s expression, plenty of siRNA therapeutic pipelines have been established by tens of biotechnology companies. The drug performance of siRNA heavily depends on the sequence, the chemical modification, and the delivery of siRNA. Here, we describe the rational design protocol of siRNA, and provide some modification patterns that can enhance siRNA’s stability and reduce its off-target effect. Also, the delivery method based on N-acetylgalactosamine (GalNAc)-siRNA conjugate that is widely employed to develop therapeutic regimens for liver-related diseases is also recapitulated." @default.
- W3157717776 created "2021-05-10" @default.
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- W3157717776 date "2021-01-01" @default.
- W3157717776 modified "2023-10-16" @default.
- W3157717776 title "siRNA Design and GalNAc-Empowered Hepatic Targeted Delivery" @default.
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- W3157717776 doi "https://doi.org/10.1007/978-1-0716-1298-9_6" @default.
- W3157717776 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/33928571" @default.
- W3157717776 hasPublicationYear "2021" @default.
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