Yoshitsugu Aoki Department of Molecular Therapy, National Institute of Neuroscience National Center of Neurology and Psychiatry Biography Publications Institution JoVE Articles Yoshitsugu Aoki has not added a biography. If you are Yoshitsugu Aoki and would like to personalize this page please email our Author Liaison for assistance. Publications Development of LNA Gapmer Oligonucleotide-Based Therapy for ALS/FTD Caused by the C9orf72 Repeat Expansion Methods in Molecular Biology (Clifton, N.J.). 2020 | Pubmed ID: 32865792 Novel EGFP Reporter Cell and Mouse Models for Sensitive Imaging and Quantification of Exon Skipping Scientific Reports. Jun, 2020 | Pubmed ID: 32572084 Severe Cardiac Involvement with Preserved Truncated Dystrophin Expression in Becker Muscular Dystrophy by +1G>A DMD Splice-site Mutation: a Case Report Journal of Human Genetics. Oct, 2020 | Pubmed ID: 32504006 Publisher Correction: Modelling Duchenne Muscular Dystrophy in MYOD1-converted Urine-derived Cells Treated with 3-deazaneplanocin A Hydrochloride Scientific Reports. Feb, 2020 | Pubmed ID: 32034287 Application of Urine-Derived Stem Cells to Cellular Modeling in Neuromuscular and Neurodegenerative Diseases Frontiers in Molecular Neuroscience. 2019 | Pubmed ID: 31920531 Amelioration of Intracellular Ca Regulation by Exon-45 Skipping in Duchenne Muscular Dystrophy-induced Pluripotent Stem Cell-derived Cardiomyocytes Biochemical and Biophysical Research Communications. Nov, 2019 | Pubmed ID: 31585729 Potential Therapies Using Myogenic Stem Cells Combined with Bio-Engineering Approaches for Treatment of Muscular Dystrophies Cells. 09, 2019 | Pubmed ID: 31514443 Exons 45-55 Skipping Using Mutation-Tailored Cocktails of Antisense Morpholinos in the DMD Gene Molecular Therapy : the Journal of the American Society of Gene Therapy. 11, 2019 | Pubmed ID: 31416775 Autoimmune Response and Its Long-term Consequences After Exon-skipping Therapy in a Duchenne Muscular Dystrophy Mouse Model The Journal of Pathology. 11, 2019 | Pubmed ID: 31322741 Peptide-conjugate Antisense Based Splice-correction for Duchenne Muscular Dystrophy and Other Neuromuscular Diseases EBioMedicine. Jul, 2019 | Pubmed ID: 31257147 Modelling Duchenne Muscular Dystrophy in MYOD1-converted Urine-derived Cells Treated with 3-deazaneplanocin A Hydrochloride Scientific Reports. 03, 2019 | Pubmed ID: 30846748 Scavenger Receptor Class A1 Mediates Uptake of Morpholino Antisense Oligonucleotide into Dystrophic Skeletal Muscle Molecular Therapy. Nucleic Acids. Mar, 2019 | Pubmed ID: 30763772 Restoring Dystrophin Expression in Duchenne Muscular Dystrophy: Current Status of Therapeutic Approaches Journal of Personalized Medicine. Jan, 2019 | Pubmed ID: 30621068 Efficacy of Multi-exon Skipping Treatment in Duchenne Muscular Dystrophy Dog Model Neonates Molecular Therapy : the Journal of the American Society of Gene Therapy. 01, 2019 | Pubmed ID: 30448197 Accelerometric Outcomes of Motor Function Related to Clinical Evaluations and Muscle Involvement in Dystrophic Dogs PloS One. 2018 | Pubmed ID: 30533017 Truncated Dystrophin Ameliorates the Dystrophic Phenotype of Mdx Mice by Reducing Sarcolipin-mediated SERCA Inhibition Biochemical and Biophysical Research Communications. 10, 2018 | Pubmed ID: 30236982 Exon Skipping Using Antisense Oligonucleotides for Laminin-Alpha2-Deficient Muscular Dystrophy Methods in Molecular Biology (Clifton, N.J.). 2018 | Pubmed ID: 30171567 In Vivo Evaluation of Multiple Exon Skipping with Peptide-PMOs in Cardiac and Skeletal Muscles in Dystrophic Dogs Methods in Molecular Biology (Clifton, N.J.). 2018 | Pubmed ID: 30171554 In Vivo Evaluation of Single-Exon and Multiexon Skipping in Mdx52 Mice Methods in Molecular Biology (Clifton, N.J.). 2018 | Pubmed ID: 30171548 In Vitro Multiexon Skipping by Antisense PMOs in Dystrophic Dog and Exon 7-Deleted DMD Patient Methods in Molecular Biology (Clifton, N.J.). 2018 | Pubmed ID: 30171540 Antisense PMO Cocktails Effectively Skip Dystrophin Exons 45-55 in Myotubes Transdifferentiated from DMD Patient Fibroblasts PloS One. 2018 | Pubmed ID: 29771942 Systemic Administration of the Antisense Oligonucleotide NS-065/NCNP-01 for Skipping of Exon 53 in Patients with Duchenne Muscular Dystrophy Science Translational Medicine. 04, 2018 | Pubmed ID: 29669851 Exon Skipping Therapy Using Phosphorodiamidate Morpholino Oligomers in the Mdx52 Mouse Model of Duchenne Muscular Dystrophy Methods in Molecular Biology (Clifton, N.J.). 2018 | Pubmed ID: 29067660 Solid-Phase Synthesis of Difficult Purine-Rich PNAs Through Selective Hmb Incorporation: Application to the Total Synthesis of Cell Penetrating Peptide-PNAs Frontiers in Chemistry. 2017 | Pubmed ID: 29094037 Quantitative Antisense Screening and Optimization for Exon 51 Skipping in Duchenne Muscular Dystrophy Molecular Therapy : the Journal of the American Society of Gene Therapy. Nov, 2017 | Pubmed ID: 28865998 Effects of Systemic Multiexon Skipping with Peptide-conjugated Morpholinos in the Heart of a Dog Model of Duchenne Muscular Dystrophy Proceedings of the National Academy of Sciences of the United States of America. Apr, 2017 | Pubmed ID: 28373570 Systemic Delivery of Morpholinos to Skip Multiple Exons in a Dog Model of Duchenne Muscular Dystrophy Methods in Molecular Biology (Clifton, N.J.). 2017 | Pubmed ID: 28364245 C9orf72 and RAB7L1 Regulate Vesicle Trafficking in Amyotrophic Lateral Sclerosis and Frontotemporal Dementia Brain : a Journal of Neurology. Apr, 2017 | Pubmed ID: 28334866 Endogenous Multiple Exon Skipping and Back-Splicing at the DMD Mutation Hotspot International Journal of Molecular Sciences. Oct, 2016 | Pubmed ID: 27754374 Anti-inflammatory Drugs for Duchenne Muscular Dystrophy: Focus on Skeletal Muscle-releasing Factors Drug Design, Development and Therapy. 2016 | Pubmed ID: 27621596 Recent Advances in Innovative Therapeutic Approaches for Duchenne Muscular Dystrophy: from Discovery to Clinical Trials American Journal of Translational Research. 2016 | Pubmed ID: 27398133 Deletion of Exons 3-9 Encompassing a Mutational Hot Spot in the DMD Gene Presents an Asymptomatic Phenotype, Indicating a Target Region for Multiexon Skipping Therapy Journal of Human Genetics. Mar, 2016 | Pubmed ID: 27009627 Serum Osteopontin As a Novel Biomarker for Muscle Regeneration in Duchenne Muscular Dystrophy The American Journal of Pathology. May, 2016 | Pubmed ID: 26963343 Self-Assembly into Nanoparticles Is Essential for Receptor Mediated Uptake of Therapeutic Antisense Oligonucleotides Nano Letters. Jul, 2015 | Pubmed ID: 26042553 Oligonucleotide Therapies: the Future of Amyotrophic Lateral Sclerosis Treatment? Neurodegenerative Disease Management. 2015 | Pubmed ID: 25894871 Long-term Efficacy of Systemic Multiexon Skipping Targeting Dystrophin Exons 45-55 with a Cocktail of Vivo-morpholinos in Mdx52 Mice Molecular Therapy. Nucleic Acids. 2015 | Pubmed ID: 25647512 Three Novel Serum Biomarkers, MiR-1, MiR-133a, and MiR-206 for Limb-girdle Muscular Dystrophy, Facioscapulohumeral Muscular Dystrophy, and Becker Muscular Dystrophy Environmental Health and Preventive Medicine. Nov, 2014 | Pubmed ID: 25150707 Oligonucleotide-Based Therapy for FTD/ALS Caused by the C9orf72 Repeat Expansion: A Perspective Journal of Nucleic Acids. 2013 | Pubmed ID: 24349764 Development of Multiexon Skipping Antisense Oligonucleotide Therapy for Duchenne Muscular Dystrophy BioMed Research International. 2013 | Pubmed ID: 23984357 Extracellular MicroRNAs Are Dynamic Non-vesicular Biomarkers of Muscle Turnover Nucleic Acids Research. Nov, 2013 | Pubmed ID: 23945935 Mutation Types and Aging Differently Affect Revertant Fiber Expansion in Dystrophic Mdx and Mdx52 Mice PloS One. 2013 | Pubmed ID: 23894429 Highly Efficient in Vivo Delivery of PMO into Regenerating Myotubes and Rescue in Laminin-α2 Chain-null Congenital Muscular Dystrophy Mice Human Molecular Genetics. Dec, 2013 | Pubmed ID: 23882132 Identification of Disease Specific Pathways Using in Vivo SILAC Proteomics in Dystrophin Deficient Mdx Mouse Molecular & Cellular Proteomics : MCP. May, 2013 | Pubmed ID: 23297347 Extensive and Prolonged Restoration of Dystrophin Expression with Vivo-morpholino-mediated Multiple Exon Skipping in Dystrophic Dogs Nucleic Acid Therapeutics. Oct, 2012 | Pubmed ID: 22888777 Bodywide Skipping of Exons 45-55 in Dystrophic Mdx52 Mice by Systemic Antisense Delivery Proceedings of the National Academy of Sciences of the United States of America. Aug, 2012 | Pubmed ID: 22869723 Identification of Muscle-specific MicroRNAs in Serum of Muscular Dystrophy Animal Models: Promising Novel Blood-based Markers for Muscular Dystrophy PloS One. 2011 | Pubmed ID: 21479190 Synthesis of 2'-O-[2-(N-methylcarbamoyl)ethyl]ribonucleosides Using Oxa-Michael Reaction and Chemical and Biological Properties of Oligonucleotide Derivatives Incorporating These Modified Ribonucleosides The Journal of Organic Chemistry. May, 2011 | Pubmed ID: 21425877 In-frame Dystrophin Following Exon 51-skipping Improves Muscle Pathology and Function in the Exon 52-deficient Mdx Mouse Molecular Therapy : the Journal of the American Society of Gene Therapy. Nov, 2010 | Pubmed ID: 20823833 Antisense PMO Found in Dystrophic Dog Model Was Effective in Cells from Exon 7-deleted DMD Patient PloS One. 2010 | Pubmed ID: 20805873 [Videofluorographic Evaluation of Dysphagia in a Patient with Myasthenia Gravis] Rinshō Shinkeigaku = Clinical Neurology. Oct, 2007 | Pubmed ID: 18095502 Relationship Between Diffusion Tensor Imaging and Brain Morphology in Patients with Myotonic Dystrophy Neuroscience Letters. Oct, 2006 | Pubmed ID: 16978781 Caracterización de exón omitiendo la eficiencia en muestras de pacientes dmd en ensayos clínicos de oligonucleótidos antisales Joel Z. Nordin*1, Yoshitaka Mizobe*1, Harumasa Nakamura2, Hirofumi Komaki3, Shin'ichi Takeda1, Yoshitsugu Aoki1 1Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, 2Clinical Research Support Office, Translational Medical Center, National Center of Neurology and Psychiatry, 3Department of Child Neurology, National Center Hospital, National Center of Neurology and Psychiatry JoVE 60672 Medicine Exon Skiping en Miotubos Directamente Reprogramados obtenidos de Células Derivadas de orina humana Hotake Takizawa1, Mitsuto Sato1, Yoshitsugu Aoki1 1Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry JoVE 60840 Genetics Multi-omisión de exón utilizando oligonucleótidos antisentido de cóctel en la distrofia muscular ligada al cromosoma X canina Bailey Miskew Nichols*1, Yoshitsugu Aoki*2, Mutsuki Kuraoka2, Joshua J.A. Lee1, Shin'ichi Takeda2, Toshifumi Yokota1 1Department of Medical Genetics, University of Alberta Faculty of Medicine and Dentistry, 2Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry JoVE 53776 Medicine
Caracterización de exón omitiendo la eficiencia en muestras de pacientes dmd en ensayos clínicos de oligonucleótidos antisales Joel Z. Nordin*1, Yoshitaka Mizobe*1, Harumasa Nakamura2, Hirofumi Komaki3, Shin'ichi Takeda1, Yoshitsugu Aoki1 1Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, 2Clinical Research Support Office, Translational Medical Center, National Center of Neurology and Psychiatry, 3Department of Child Neurology, National Center Hospital, National Center of Neurology and Psychiatry JoVE 60672 Medicine
Exon Skiping en Miotubos Directamente Reprogramados obtenidos de Células Derivadas de orina humana Hotake Takizawa1, Mitsuto Sato1, Yoshitsugu Aoki1 1Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry JoVE 60840 Genetics
Multi-omisión de exón utilizando oligonucleótidos antisentido de cóctel en la distrofia muscular ligada al cromosoma X canina Bailey Miskew Nichols*1, Yoshitsugu Aoki*2, Mutsuki Kuraoka2, Joshua J.A. Lee1, Shin'ichi Takeda2, Toshifumi Yokota1 1Department of Medical Genetics, University of Alberta Faculty of Medicine and Dentistry, 2Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry JoVE 53776 Medicine
