Toshifumi Yokota
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Computer Science
Toshifumi Yokota's Degrees
- PhD Computer Science University of Tokyo
- Masters Computer Science University of Tokyo
- Bachelors Computer Science University of Tokyo
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Why Is Toshifumi Yokota Influential?
(Suggest an Edit or Addition)According to Wikipedia, Toshifumi Yokota is a medical scientist and professor of medical genetics at the University of Alberta, where he also holds the titles of the Friends of Garrett Cumming Research & Muscular Dystrophy Canada Endowed Research Chair and the Henri M. Toupin Chair in Neurological Science. He is best known for his studies of antisense oligonucleotide-based therapeutics for muscular dystrophy that led to the development of an FDA-approved drug viltolarsen. His research interests include precision medicine for muscular dystrophy and genetic diseases. He has co-edited two books both published in the Methods in Molecular Biology series from Humana Press, Springer-Nature, and has published more than 100 refereed papers and patents. He is a member of the editorial boards for the International Journal of Molecular Sciences, Genes, Frontiers in Genome Editing, Frontiers in Physiology, and Nucleic Acid Therapeutics, a member of the Medical and Scientific Advisory Committee of Muscular Dystrophy Canada, and a co-founder of the Canadian Neuromuscular Network .
Toshifumi Yokota's Published Works
Number of citations in a given year to any of this author's works
Total number of citations to an author for the works they published in a given year. This highlights publication of the most important work(s) by the author
Published Works
- Eteplirsen in the treatment of Duchenne muscular dystrophy (2017) (515)
- Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles. (2005) (393)
- Efficacy of systemic morpholino exon‐skipping in duchenne dystrophy dogs (2009) (378)
- Bodywide skipping of exons 45–55 in dystrophic mdx52 mice by systemic antisense delivery (2012) (137)
- Pathogenic exon-trapping by SVA retrotransposon and rescue in Fukuyama muscular dystrophy (2011) (135)
- The status of exon skipping as a therapeutic approach to duchenne muscular dystrophy. (2011) (107)
- α1-Syntrophin Modulates Turnover of ABCA1* (2004) (101)
- In-frame dystrophin following exon 51-skipping improves muscle pathology and function in the exon 52-deficient mdx mouse. (2010) (97)
- Expansion of revertant fibers in dystrophic mdx muscles reflects activity of muscle precursor cells and serves as an index of muscle regeneration (2006) (97)
- Restoring dystrophin expression in duchenne muscular dystrophy muscle progress in exon skipping and stop codon read through. (2011) (96)
- Micro-dystrophin cDNA ameliorates dystrophic phenotypes when introduced into mdx mice as a transgene. (2002) (94)
- Effects of systemic multiexon skipping with peptide-conjugated morpholinos in the heart of a dog model of Duchenne muscular dystrophy (2017) (83)
- α1-Syntrophin–deficient skeletal muscle exhibits hypertrophy and aberrant formation of neuromuscular junctions during regeneration (2002) (74)
- Extensive and prolonged restoration of dystrophin expression with vivo-morpholino-mediated multiple exon skipping in dystrophic dogs. (2012) (73)
- Long-Term Efficacy of Systemic Multiexon Skipping Targeting Dystrophin Exons 45–55 With a Cocktail of Vivo-Morpholinos in Mdx52 Mice (2015) (70)
- Restoring Dystrophin Expression in Duchenne Muscular Dystrophy: Current Status of Therapeutic Approaches (2019) (70)
- Optimizing exon skipping therapies for DMD. (2007) (65)
- Quantitative Antisense Screening and Optimization for Exon 51 Skipping in Duchenne Muscular Dystrophy. (2017) (62)
- A renaissance for antisense oligonucleotide drugs in neurology: exon skipping breaks new ground. (2009) (61)
- Skipping Multiple Exons to Treat DMD—Promises and Challenges (2018) (59)
- Highly efficient in vivo delivery of PMO into regenerating myotubes and rescue in laminin-α2 chain-null congenital muscular dystrophy mice (2013) (58)
- Antisense PMO Found in Dystrophic Dog Model Was Effective in Cells from Exon 7-Deleted DMD Patient (2010) (58)
- Antisense Therapy in Neurology (2013) (58)
- Aquaporin-4 is absent at the sarcolemma and at perivascular astrocyte endfeet in α1-syntrophin knockout mice (2000) (56)
- Potential of oligonucleotide-mediated exon-skipping therapy for Duchenne muscular dystrophy (2007) (54)
- Multiple Exon Skipping in the Duchenne Muscular Dystrophy Hot Spots: Prospects and Challenges (2018) (54)
- Alpha1-syntrophin modulates turnover of ABCA1. (2004) (54)
- Skipping multiple exons of dystrophin transcripts using cocktail antisense oligonucleotides. (2014) (53)
- New developments in exon skipping and splice modulation therapies for neuromuscular diseases (2014) (53)
- Dystrophin-deficient large animal models: translational research and exon skipping. (2015) (51)
- Development of Multiexon Skipping Antisense Oligonucleotide Therapy for Duchenne Muscular Dystrophy (2013) (47)
- Current Translational Research and Murine Models For Duchenne Muscular Dystrophy (2016) (47)
- Optimization of antisense-mediated exon skipping for Duchenne muscular dystrophy (2020) (46)
- In Silico Screening Based on Predictive Algorithms as a Design Tool for Exon Skipping Oligonucleotides in Duchenne Muscular Dystrophy (2015) (46)
- Applications of CRISPR/Cas9 for the Treatment of Duchenne Muscular Dystrophy (2018) (46)
- Antisense oligo-mediated multiple exon skipping in a dog model of duchenne muscular dystrophy. (2011) (40)
- Comparison of the phenotypes of patients harboring in-frame deletions starting at exon 45 in the Duchenne muscular dystrophy gene indicates potential for the development of exon skipping therapy (2016) (40)
- Viltolarsen for the treatment of Duchenne muscular dystrophy. (2019) (40)
- An Overview of Recent Advances and Clinical Applications of Exon Skipping and Splice Modulation for Muscular Dystrophy and Various Genetic Diseases. (2018) (39)
- Nonmechanical Roles of Dystrophin and Associated Proteins in Exercise, Neuromuscular Junctions, and Brains (2015) (36)
- 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 (2016) (35)
- Impaired regenerative capacity and lower revertant fibre expansion in dystrophin-deficient mdx muscles on DBA/2 background (2016) (34)
- Muscle dysfunction caused by loss of Magel2 in a mouse model of Prader-Willi and Schaaf-Yang syndromes. (2016) (33)
- Exon skipping for nonsense mutations in Duchenne muscular dystrophy: too many mutations, too few patients? (2012) (33)
- Golodirsen for Duchenne muscular dystrophy. (2020) (32)
- LNA/DNA mixmer-based antisense oligonucleotides correct alternative splicing of the SMN2 gene and restore SMN protein expression in type 1 SMA fibroblasts (2017) (32)
- DUX4 Signalling in the Pathogenesis of Facioscapulohumeral Muscular Dystrophy (2020) (31)
- Inhibition of DUX4 expression with antisense LNA gapmers as a therapy for facioscapulohumeral muscular dystrophy (2020) (31)
- Antisense oligonucleotides for the treatment of cardiomyopathy in Duchenne muscular dystrophy. (2019) (28)
- Developing DMD therapeutics: a review of the effectiveness of small molecules, stop-codon readthrough, dystrophin gene replacement, and exon-skipping therapies (2021) (28)
- Immortalized Muscle Cell Model to Test the Exon Skipping Efficacy for Duchenne Muscular Dystrophy (2017) (27)
- Mutation Types and Aging Differently Affect Revertant Fiber Expansion in Dystrophic Mdx and Mdx52 Mice (2013) (26)
- Designing Effective Antisense Oligonucleotides for Exon Skipping. (2018) (26)
- Exons 45-55 Skipping Using Mutation-Tailored Cocktails of Antisense Morpholinos in the DMD Gene. (2019) (24)
- A novel human muscle cell model of Duchenne muscular dystrophy created by CRISPR/Cas9 and evaluation of antisense-mediated exon skipping (2018) (24)
- Invention and Early History of Exon Skipping and Splice Modulation. (2018) (22)
- Current understanding and treatment of cardiac and skeletal muscle pathology in laminin-α2 chain-deficient congenital muscular dystrophy (2019) (22)
- Multi-exon Skipping Using Cocktail Antisense Oligonucleotides in the Canine X-linked Muscular Dystrophy (2016) (22)
- Antisense oligonucleotide drugs for Duchenne muscular dystrophy: how far have we come and what does the future hold? (2015) (22)
- Efficacy of Multi-exon Skipping Treatment in Duchenne Muscular Dystrophy Dog Model Neonates. (2019) (21)
- “Of Mice and Measures”: A Project to Improve How We Advance Duchenne Muscular Dystrophy Therapies to the Clinic (2018) (20)
- Antisense PMO cocktails effectively skip dystrophin exons 45-55 in myotubes transdifferentiated from DMD patient fibroblasts (2018) (20)
- Scavenger Receptor Class A1 Mediates Uptake of Morpholino Antisense Oligonucleotide into Dystrophic Skeletal Muscle (2019) (19)
- Nusinersen in the Treatment of Spinal Muscular Atrophy. (2018) (19)
- Morpholino treatment improves muscle function and pathology of Pitx1 transgenic mice. (2014) (18)
- Cardiac Involvement in Dystrophin-Deficient Females: Current Understanding and Implications for the Treatment of Dystrophinopathies (2020) (17)
- Systemic Delivery of Morpholinos to Skip Multiple Exons in a Dog Model of Duchenne Muscular Dystrophy. (2017) (16)
- Identification of Novel Antisense-Mediated Exon Skipping Targets in DYSF for Therapeutic Treatment of Dysferlinopathy (2018) (16)
- Recent advancements in exon-skipping therapies using antisense oligonucleotides and genome editing for the treatment of various muscular dystrophies (2019) (16)
- Knocking Down Long Noncoding RNAs Using Antisense Oligonucleotide Gapmers. (2020) (16)
- Genome Editing for the Understanding and Treatment of Inherited Cardiomyopathies (2020) (16)
- Advances in Genetic Characterization and Genotype–Phenotype Correlation of Duchenne and Becker Muscular Dystrophy in the Personalized Medicine Era (2020) (14)
- Genotype–Phenotype Correlations in Duchenne and Becker Muscular Dystrophy Patients from the Canadian Neuromuscular Disease Registry (2020) (13)
- CRISPR-Generated Animal Models of Duchenne Muscular Dystrophy (2020) (13)
- Pharmacology and toxicology of eteplirsen and SRP-5051 for DMD exon 51 skipping: an update (2021) (13)
- Restoring SMN Expression: An Overview of the Therapeutic Developments for the Treatment of Spinal Muscular Atrophy (2022) (13)
- DUX4 transcript knockdown with antisense 2'-O-methoxyethyl gapmers for the treatment of facioscapulohumeral muscular dystrophy. (2020) (13)
- Exon Skipping Therapy Using Phosphorodiamidate Morpholino Oligomers in the mdx52 Mouse Model of Duchenne Muscular Dystrophy. (2018) (12)
- Amelioration of intracellular Ca2+ regulation by exon-45 skipping in Duchenne muscular dystrophy-induced pluripotent stem cell-derived cardiomyocytes. (2019) (11)
- Mutation-independent Proteomic Signatures of Pathological Progression in Murine Models of Duchenne Muscular Dystrophy (2020) (11)
- Membrane Repair Deficit in Facioscapulohumeral Muscular Dystrophy (2020) (11)
- Antisense and Gene Therapy Options for Duchenne Muscular Dystrophy Arising from Mutations in the N-Terminal Hotspot (2022) (11)
- Development of DG9 peptide-conjugated single- and multi-exon skipping therapies for the treatment of Duchenne muscular dystrophy (2022) (9)
- Casimersen for Duchenne muscular dystrophy. (2021) (9)
- Recent Advances and Clinical Applications of Exon Inclusion for Spinal Muscular Atrophy. (2018) (9)
- Invention and Early History of Gapmers. (2020) (9)
- Inotersen for the Treatment of Hereditary Transthyretin Amyloidosis. (2020) (9)
- Translational Research in Nucleic Acid Therapies for Muscular Dystrophies (2016) (8)
- Development of Antisense Oligonucleotide Gapmers for the Treatment of Dyslipidemia and Lipodystrophy. (2020) (8)
- CRISPR Therapeutics for Duchenne Muscular Dystrophy (2022) (8)
- eSkip-Finder: a machine learning-based web application and database to identify the optimal sequences of antisense oligonucleotides for exon skipping (2021) (8)
- Creation of DMD Muscle Cell Model Using CRISPR-Cas9 Genome Editing to Test the Efficacy of Antisense-Mediated Exon Skipping. (2018) (8)
- Targeting mRNA for the treatment of facioscapulohumeral muscular dystrophy. (2016) (7)
- Applications of CRISPR / Cas 9 for the Treatment of Duchenne Muscular Dystrophy (2019) (7)
- Restoring Protein Expression in Neuromuscular Conditions: A Review Assessing the Current State of Exon Skipping/Inclusion and Gene Therapies for Duchenne Muscular Dystrophy and Spinal Muscular Atrophy (2021) (7)
- Muscular Dystrophy: Disease Mechanisms and Therapies (2015) (6)
- Tips to Design Effective Splice-Switching Antisense Oligonucleotides for Exon Skipping and Exon Inclusion. (2018) (6)
- Development and Clinical Applications of Antisense Oligonucleotide Gapmers. (2020) (6)
- 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. (2018) (6)
- Development of Antisense Oligonucleotide Gapmers for the Treatment of Huntington's Disease. (2020) (6)
- Immortalized Canine Dystrophic Myoblast Cell Lines for Development of Peptide-Conjugated Splice-Switching Oligonucleotides (2021) (6)
- α1‐Syntrophin–deficient mice exhibit impaired muscle force recovery after osmotic shock (2014) (6)
- A Genotype-Phenotype Correlation Study of Exon Skip-Equivalent In-Frame Deletions and Exon Skip-Amenable Out-of-Frame Deletions across the DMD Gene to Simulate the Effects of Exon-Skipping Therapies: A Meta-Analysis (2021) (5)
- Pharmacological Profile of Viltolarsen for the Treatment of Duchenne Muscular Dystrophy: A Japanese Experience (2021) (5)
- Genetic Approaches for the Treatment of Facioscapulohumeral Muscular Dystrophy (2021) (5)
- A Dystrophin Exon-52 Deleted Miniature Pig Model of Duchenne Muscular Dystrophy and Evaluation of Exon Skipping (2021) (5)
- In Vivo Evaluation of Multiple Exon Skipping with Peptide-PMOs in Cardiac and Skeletal Muscles in Dystrophic Dogs. (2018) (5)
- Systemic Injection of Peptide-PMOs into Humanized DMD Mice and Evaluation by RT-PCR and ELISA. (2018) (4)
- Degradation of Toxic RNA in Myotonic Dystrophy Using Gapmer Antisense Oligonucleotides. (2020) (4)
- Cell Membrane Repair Assay Using a Two-photon Laser Microscope. (2018) (4)
- Restoration of dystrophin expression and correction of Duchenne muscular dystrophy by genome editing (2021) (4)
- Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts. (2018) (4)
- Exon Skipping Using Antisense Oligonucleotides for Laminin-Alpha2-Deficient Muscular Dystrophy. (2018) (3)
- In Vitro Multiexon Skipping by Antisense PMOs in Dystrophic Dog and Exon 7-Deleted DMD Patient. (2018) (2)
- Morpholino-Mediated Exon Skipping Targeting Human ACVR1/ALK2 for Fibrodysplasia Ossificans Progressiva. (2018) (2)
- In Vivo Evaluation of Single-Exon and Multiexon Skipping in mdx52 Mice. (2018) (2)
- Methods of CRISPR/Cas9 Exon Skipping for Duchenne Muscular Dystrophy (2018) (2)
- Molecular Diagnosis and Novel Therapies for Neuromuscular Diseases (2020) (2)
- Potential of antisense therapy for facioscapulohumeral muscular dystrophy (2015) (1)
- Cardio‐respiratory and phenotypic rescue of dystrophin/utrophin‐deficient mice by combination therapy (2022) (1)
- Allele-Selective Locked Nucleic Acid Gapmers for the Treatment of Fibrodysplasia Ossificans Progressiva Knock Down the Pathogenic ACVR1R206H Transcript and Inhibit Osteogenic Differentiation. (2022) (1)
- Quantitative Evaluation of Exon Skipping in Immortalized Muscle Cells In Vitro. (2018) (1)
- Systemic and ICV Injections of Antisense Oligos into SMA Mice and Evaluation. (2018) (1)
- Mutation-independent Proteomic Signatures of Pathological Progression in Murine Models of Duchenne Muscular Dystrophy. (2020) (1)
- Biological and genetic therapies for the treatment of Duchenne muscular dystrophy (2022) (1)
- In Vitro Evaluation of Antisense-Mediated Exon Inclusion for Spinal Muscular Atrophy. (2018) (1)
- Exon Skipping and Inclusion Therapies (2018) (1)
- DG9-conjugated morpholino rescues phenotype in SMA mice by reaching the CNS via a subcutaneous administration (2023) (1)
- D02 Age-related and mutation-independent proteomic changes in dystrophic mouse muscle (2017) (0)
- 623. Dystrophin Exon 52-Deleted Pigs as a New Animal Model of Duchenne Muscular Dystrophy: Its Characterization and Potential as a Tool for Developing Exon Skipping Therapy (2016) (0)
- T.O.2 Systemic delivery of morpholino oligonucleotides to skip mutations in the dystrophin gene of the mouse and dog (2007) (0)
- Editorial: Genome and transcriptome editing to understand and treat neuromuscular diseases (2023) (0)
- A novel human muscle cell model of Duchenne muscular dystrophy created by CRISPR/Cas9 and evaluation of antisense-mediated exon skipping (2018) (0)
- Detection of Locked Nucleic Acid Gapmers from Mouse Muscle Samples Using ELISA. (2020) (0)
- Exons 45-55 Skipping Using Antisense Oligonucleotides in Immortalized Human DMD Muscle Cells. (2023) (0)
- Gapmers (2020) (0)
- Genome Editing for the Understanding and Treatment of Cardiomyopathies (2019) (0)
- The exon 45 skipping therapy of induced pluripotent stem cells derived cardiomyocyte from the DMD patient with exon 46-55 deletion (2017) (0)
- DMD – ANIMAL MODELS & PRECLINICAL TREATMENT P.214 CRISPR-Cas9 genome editing rescues dystrophin expression in a dog DMD model with a mutation in the N-terminal mutation hotspot (2020) (0)
- Current Strategies of Muscular Dystrophy Therapeutics: An Overview. (2023) (0)
- Genetic Approaches for the Treatment of Giant Axonal Neuropathy (2022) (0)
- Natural History of a Mouse Model Overexpressing the Dp71 Dystrophin Isoform (2021) (0)
- In Vivo Evaluation of Exon 51 Skipping in hDMD/Dmd-null Mice. (2023) (0)
- Methods of CRISPR / Cas 9 exon skipping for Duchenne muscular dystrophy (2018) (0)
- 378. An AAV Vector-Mediated Micro-Dystrophin Expression in Relatively Small Percentage of Dystrophin-Deficient mdx Myofibers Still Improved the mdx Phenotype Through Compensatory Hypertrophy (2004) (0)
- Revertant fiber studies in Duchenne muscular dystrophy (2013) (0)
- P3.04 Skipping of exons 6 and 8 of the DMD gene has been achieved in myogenic cells from an exon-7 deleted DMD patient: direct application of antisense sequences found in study with canine muscular dystrophy (2010) (0)
- Generation of clusters of dystrophin revertant fibers requires muscle regeneration and tests the function of dystrophin/utrophin transgenes (2004) (0)
- Rapid Freezing of Skeletal and Cardiac Muscles Using Isopentane Cooled with Liquid Nitrogen and Tragacanth Gum for Histological, Genetic, and Protein Expression Studies. (2023) (0)
- Enhancing the Effectiveness of Oligonucleotide Therapeutics Using Cell-Penetrating Peptide Conjugation, Chemical Modification, and Carrier-Based Delivery Strategies (2023) (0)
- Antisense Oligonucleotide Treatment in a Humanized Mouse Model of Duchenne Muscular Dystrophy and Highly Sensitive Detection of Dystrophin Using Western Blotting. (2021) (0)
- Optimization of antisense-mediated exon skipping for Duchenne muscular dystrophy (2020) (0)
- Recent Trends in Antisense Therapies for Duchenne Muscular Dystrophy (2023) (0)
- DEVELOPING AN EFFECTIVE EXONS 45-55 SKIPPING THERAPY FOR DUCHENNE MUSCULAR DYSTROPHY (2020) (0)
- Assessing the Role of Aquaporin 4 in Skeletal Muscle Function (2023) (0)
- Dystrophin-deficient cardiomyocyte derived from Duchenne Muscular Dystrophy specific induced pluripotent stem cells carrying the deletion of exon 46-55 in DMD gene (2017) (0)
- Knocking Down DUX4 in Immortalized Facioscapulohumeral Muscular Dystrophy Patient-Derived Muscle Cells. (2023) (0)
- Related articles (2013) (0)
- CPAA_A_288842 235..242 (2021) (0)
- Viltolarsen: From Preclinical Studies to FDA Approval. (2023) (0)
- Restoring Dystrophin Expression by Skipping Exons 6 and 8 in Neonatal Dystrophic Dogs. (2023) (0)
- Morpholino-Mediated Exons 28-29 Skipping of Dysferlin and Characterization of Multiexon-skipped Dysferlin using RT-PCR, Immunoblotting, and Membrane Wounding Assay. (2023) (0)
- Construction of a novel Duchenne muscular dystrophy model cell line with CRISPR/Cas9 system (2016) (0)
- LNA/DNA mixmer-based antisense oligonucleotides correct alternative splicing of the SMN2 gene and restore SMN protein expression in type 1 SMA fibroblasts (2017) (0)
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