Exon Skipping and Inclusion Therapies

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 toTest 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 forCombinatorial 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.