Exon Skipping and Inclusion Therapies

Methods and Protocols

Inbunden, Engelska, 2025

Av Yusuke Echigoya, Rika Maruyama, Toshifumi Yokota

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Produktinformation

Utgivningsdatum2025-07-29
Mått178 x 254 x 34 mm
Vikt1 163 g
FormatInbunden
SpråkEngelska
SerieMethods in Molecular Biology
Antal sidor503
Upplaga2
FörlagSpringer-Verlag New York Inc.
ISBN9781071647295

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Medicinsk genetik inom Medicin
Sjukdomar och rubbningar inom Medicin
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Progress and Future Directions in Exon Skipping and Inclusion Therapies: The Landscape of Oligonucleotide-Based Genetic Medicine.- Evolution and Breakthroughs in Exon Skipping and Splice Modulation: From Inception to Clinical Success.- An Overview of Recent Advances and Clinical Applications of Exon Skipping and Splice Modulation for Muscular Dystrophy and Various Genetic Diseases.- Milasen: The Emerging Era of Patient-Customized N-of-1 Antisense Oligonucleotides as Therapeutic Agents for Genetic Diseases.- Tips to Design Effective Splice-Switching Antisense Oligonucleotides for Exon Skipping and Exon Inclusion.- In Silico Prediction and Selection of Exon-Skipping Antisense Oligonucleotide Sequences Using eSkip-Finder.- Quantitative Evaluation of Exon Skipping in Immortalized Muscle Cells In Vitro.- Direct Reprogramming of Human DMD Fibroblasts into Myotubes for In Vitro Evaluation of Antisense-Mediated Exon Skipping and Exons 45-55 Skipping Accompanied by Rescue of Dystrophin Expression.- In Vitro Multiexon Skipping by Antisense PMOs in Dystrophic Dog and Exon 7-Deleted DMD Patient.- Creation of DMD Muscle Cell Model Using CRISPR-Cas9 Genome Editing to Test the Efficacy of Antisense-Mediated Exon Skipping.- In Vitro Evaluation of Exon Skipping in Disease Specific iPSC-Derived Myocytes.- Skipping of Duplicated Dystrophin Exons: In Vitro Induction and Assessment.- In Vivo Evaluation of Dystrophin Exon Skipping in mdx Mice.- Guide to Selection of Muscle-Homing Peptides after In Vivo Phage Display Biopanning.- Systemic Injection of Peptide-PMOs into Humanized DMD Mice and Detection by RT-PCR and ELISA.- In Vivo Evaluation of Single- and Multi-Exon Skipping in mdx52 Mice.- A Novel Zebrafish Model for Assessing In Vivo Delivery of Morpholino Oligomers.- Electrophysiological Evaluation in mdx52 Mouse Brain after Antisense-Mediated Exon 51 or 53 Skipping.- Use of Glucose/Fructose to Enhance the Exon Skipping Efficacy.- Systemic Intravenous Administration of Antisense Therapeutics for Combinatorial Dystrophin and Myostatin Exon Splice Modulation.- The Assembly of Fluorescently Labeled Peptide-Oligonucleotide Conjugates.- In Vivo Evaluation of Multiple Exon Skipping with Peptide-PMOs in Cardiac and Skeletal Muscles in Dystrophic Dogs.- Use of Tricyclo-DNA Antisense Oligonucleotides for Exon Skipping.- Optimization of 2ʹ,4ʹ-BNA/LNA-Based Oligonucleotides for Splicing Modulation In Vitro.- Pre-mRNA Splicing Modulation by Antisense Oligonucleotides.- In Vitro Evaluation of Antisense-Mediated Exon Inclusion for Spinal Muscular Atrophy.- Systemic Injection of Antisense Oligos into Spinal Muscular Atrophy (SMA) Mice and Evaluation.- Morpholino-Mediated Exon Inclusion for Spinal Muscular Atrophy (SMA).- Exon Skipping by Ultrasound-Enhanced Delivery of Morpholino with Bubble Liposomes for Myotonic Dystrophy Model Mice.- Restoration of Dysferlin after Exon 32 Skipping in Patient Cells.- Morpholino-Mediated Exon Skipping Targeting Human ACVR1/ALK2 for Fibrodysplasia Ossificans Progressiva.- Exon Skipping of FcεRIβ for Allergic Diseases.- Exon-Skipping Using Antisense Oligonucleotides for Laminin-Alpha2-Deficient Muscular Dystrophy.