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Titlebook: Oligonucleotide-Based Therapies; Methods and Protocol Olof Gissberg,Rula Zain,Karin E. Lundin Book 2019 Springer Science+Business Media, LL

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Chemical Development of Therapeutic Oligonucleotides. Here we discuss selected chemistries used in antisense oligonucleotide (ASO) applications (e.g., small interfering RNA (siRNA), RNase H activation, translational block, splice-switching, and also as aptamers). Recently approved oligonucleotide-based drugs are also presented briefly.
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Delivery of Antisense Oligonucleotides Mediated by a Hydrogel System: In Vitro and In Vivo Applicati Here we describe the preparation of antisense oligonucleotide-loaded fibrin hydrogels exploring their applications in the context of the nervous system utilizing an organotypic dorsal root ganglion explant in vitro system and an in vivo model of spinal cord injury.
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Evaluation of Cell-Penetrating Peptide Delivery of Antisense Oligonucleotides for Therapeutic Efficaf ASOs. One of the most advanced clinical uses of ASOs is for the treatment of spinal muscular atrophy (SMA). In this chapter, we describe the techniques used for in vitro screening and analysing in vivo biodistribution of CPP-conjugated ASOs targeting the survival motor neuron 2, ., the dose-dependent modifying gene for SMA.
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Identifying Suitable Target Regions and Analyzing Off-Target Effects of Therapeutic Oligonucleotidesprogramming language R, and the calculations and data processing can be carried out on a desktop computer. Our workflow integrates well-established data resources and RNA structure-prediction tools and can be modified easily and expanded as new resources become available.
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