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

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In Vitro Delivery of PMOs in Myoblasts by Electroporation affect its splicing gives AONs potential use for exon skipping therapies aimed at restoring the dystrophin transcript reading frame for Duchenne muscular dystrophy (DMD) patients. The neutrally charged phosphorodiamidate morpholino oligomers (PMOs) are a stable and relatively nontoxic AON modificat

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Evaluation of Exon Skipping and Dystrophin Restoration in In Vitro Models of Duchenne Muscular Dystre muscular dystrophy, but many more are in development targeting an array of different . exons. Preclinical screening of the new oligonucleotide sequences is routinely performed using patient-derived cell cultures, and evaluation of their efficacy may be performed at RNA and/or protein level. While

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Generation of Human iPSC-Derived Myotubes to Investigate RNA-Based Therapies In Vitrof canonical pre-mRNA splicing by disease-associated variants can result in genetic disorders. Antisense oligonucleotides (AONs) offer an attractive solution to modulate endogenous gene expression through alteration of pre-mRNA splicing events. Relevant in vitro models are crucial for appropriate eva

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2.1.6 References to crystal structure,RNA, synthetic SINEUP molecules have been developed by creating a specific BD for the gene of interest and placing it upstream the invSINEB2 ED. Synthetic SINEUP is thus a novel molecular tool that potentially may be used for any industrial or biomedical application to enhance protein production, al