Titlebook: Antisense RNA Design, Delivery, and Analysis; Virginia Arechavala-Gomeza,Alejandro Garanto Book‘‘‘‘‘‘‘‘ 2022 The Editor(s) (if applicable)

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Introduction and History of the Chemistry of Nucleic Acids Therapeuticsapeutics focusing in particular on antisense oligonucleotide analogues carrying modifications in the backbone and sugar. Brief mention is made of siRNA development and other applications that have by and large utilized the same modifications. We also point out the pitfalls of the use of nucleic acid

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Antisense RNA Therapeutics: A Brief OverviewRNA vaccines against SARS-CoV-2. Another type of nucleic acid therapeutics is antisense oligonucleotides, versatile tools that can be used in multiple ways to target pre-mRNA and mRNA. While some years ago these molecules were just considered a useful research tool and a curiosity in the clinical ma

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arthrodesis

Design and Delivery of SINEUP: A New Modular Tool to Increase Protein Translationessary to specifically upregulate target gene translation. Originally identified in the mouse . (antisense Ubiquitin carboxyl-terminal esterase L1) locus, natural SINEUP molecules are oriented head to head to their sense protein coding, target gene (., in this example). Peculiarly, SINEUP is able to

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How to Design U1 snRNA Molecules for Splicing RescueRNA splicing requires the correct identification of a number of .-acting elements in an ordered fashion. By disrupting the complementarity of the 5′ss with the endogenous small nuclear RNA U1 (U1 snRNA), the key component of the spliceosomal U1 ribonucleoprotein, 5′ss mutations may result in exon sk

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Conjugation of Nucleic Acids and Drugs to Gold Nanoparticleslization into the cell and prevent their degradation. In addition, engineered AuNPs can be employed as sensors of a variety of biomarkers, where the electronic and optical properties of the AuNPs are exploited for a convenient, easy, and fast read out. However, in all these applications, a key step

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Determination of Optimum Ratio of Cationic Polymers and Small Interfering RNA with Agarose Gel Retar gene delivery are positively charged to carry negatively charged oligonucleotides. However, excessive positively charged carriers are cytotoxic. Therefore, the complexed oligonucleotide/nanoparticles should be well-examined before the application. In that manner, agarose gel electrophoresis, which

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