Exon Skipping and Inclusion Therapies: Methods and Protocols Second Edition 2025 [Kõva köide]

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 2,4-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 FcRI for Allergic Diseases.- Exon-Skipping Using Antisense
Oligonucleotides for Laminin-Alpha2-Deficient Muscular Dystrophy.