Splice Switching

DG9-Conjugated Morpholino-Based Exon 51 Skipping Therapy for Duchenne Muscular Dystrophy.- DG9-Conjugated Morpholino Rescues Phenotype in Spinal Muscular Atrophy Mice.- Cardio-Physiological Evaluations in A Dog Model with Duchenne Muscular Dystrophy.- Splice Modulation Studies Using Urine-Derived Cells.- Lipidomic Sample Preparation to Detect Phosphatidylcholine Alterations in Skeletal Muscle.- Evaluation for the Efficacy of Exon Skipping Therapy Using In Vivo Assessment of Skeletal Muscle Function and Fragility.- Characterization and Phenotypic Assessment of Zebrafish Disease Models for Splice Modulation Studies.- Preparation of Size-Defined PEG-Grafted Copolymers as a Polymeric Nanoruler for Size Optimization in Passive Targeting.- Splice Modulation Studies Using Reporter Mice.- Clinical Variant Databases and Machine Learning Prediction Supporting Genomic Medicine.- In Vivo Evaluation of CTG Repeat-Affected Muscle Pathology in a Myotonic Dystrophy Model Mouse Using Electromyography and Fluorescence In Situ Hybridization.- Utilization of Induced Pluripotent Stem Cell-Derived Neurons to Investigate Splice-Modification Efficacy of Splice-Switching Drug Candidates.- Development of Electric Field Stimulation (EFS)-Mediated Skeletal Muscle Training with iPSCs.- Neurological Behavioral Assessment of Exon Skipping Therapy for Neuromuscular Disorder Using Displaced Object Recognition Test, Open Field Test, and Three-Chamber Social Approach Test.- Analysis of Prefrontal-Amygdala Synaptic Functions Using Optogenetics and Path-Clamp Recording.- Digital Outcome Measures for Gait and Spontaneous Locomotor Activity in Canine Dystrophic Model.