Titlebook: Design and Delivery of SiRNA Therapeutics; Henrik J. Ditzel,Martina Tuttolomondo,Sakari Kaupp Book 2021 Springer Science+Business Media, L

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Abbildungen, reelle Funktionen,itro and in vivo. Here we provide a robust protocol for the synthesis of triple GalNAc CPG solid support and GalNAc phosphoramidite, synthesis and purification of RNA conjugates with multiple GalNAc residues either to 5′-end or 3′-end and siRNA duplex formation.

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Wilfried Pla?mann,Detlef Schulz stability of the siRNA/NV ensemble; (3) characterization and quantification of the cellular uptake and release of the siRNA fragment; (4) in vitro and (5) in vivo experiments for the evaluation of the corresponding gene silencing activity; and (6) assessment of the intrinsic toxicity of the NV and the siRNA/NV complex.

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aesthetician

Development of siRNA Therapeutics for the Treatment of Liver Diseases,nd givosiran for the treatment of acute hepatic porphyria, respectively. Here, we describe the current strategies for delivering siRNA drugs to the liver and summarize recent advances in clinical development of siRNA therapeutics for the treatment of liver diseases.

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siRNA Design and GalNAc-Empowered Hepatic Targeted Delivery,lity and reduce its off-target effect. Also, the delivery method based on .-acetylgalactosamine (GalNAc)-siRNA conjugate that is widely employed to develop therapeutic regimens for liver-related diseases is also recapitulated.

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