Exon skipping and inclusion therapies methods and protocols - Yokota, Toshifumi ca. 20./21. Jh; Maruyama, Rika ca. 20./21. Jh (Springer New York, 2018)
Invention and Early History of Exon Skipping and Splice Modulation -- An Overview of Recent Advances and Clinical Applications of Exon Skipping and Splice Modulation for Muscular Dystrophy and Various Genetic Diseases -- Recent Advances and Clinical Applications of Exon Inclusion for Spinal Muscular Atrophy -- Nusinersen in the Treatment of Spinal Muscular Atrophy -- Tips to Design Effective Splice-switching Antisense Oligonucleotides for Exon Skipping and Exon Inclusion -- Antisense Oligonucleotide Targeting of 3'UTR of mRNA for Expression Knockdown -- 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-m Exon Skipping and Exons 45-55 Skipping Accompanied by Rescue of Dystrophin Expression -- In vitro Multi-exon 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 -- Restoration of Dystrophin Protein Expression by Exon Skipping utilizing CRISPR-Cas9 in Myoblasts Derived from DMD Patient iPS Cells -- Skipping of Duplicated Dystrophin Exons: in vitro Induction and Assessment -- In Vivo Evaluation of Dystrophin Exon Skipping in mdx Mice -- Exon 51 Skipping Quantification by Digital Droplet PCR in del52hDMD/mdx Mice -- 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 -- Validation and Detection of Exon Skipping Boosters in DMD Patient Cell Models and mdx Mouse -- 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 via Orthogonal Ligation Strategies -- 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 SMA Mice and Evaluation -- Exon Skipping using Antisense Oligonucleotides for Laminin-alpha2-deficient Muscular Dystrophy -- Exon Skipping by Ultrasound-enhanced Delivery of Morpholino with Bubble Liposomes for Myotonic Dystrophy Model Mice --
- Dysferlin 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 -- Antisense Oligonucleotide Design and Evaluation of Splice-modulating Properties Using Cell-based Assays -- Antisense-mediated Splice Modulation to Reframe Transcripts -- Morpholino-mediated Exon Inclusion for SMA.