Takanori Yokota 研究室

主宰者：Takanori Yokota

理化学研究所・RIKEN Center for Brain Science

AI 要約（直近 5 年の研究成果）

Takanori Yokota 研究室は、神経筋疾患の治療開発に向けて、核酸医薬と遺伝子制御の手法に取り組んでいます。筋ジストロフィーや脊髄性筋萎縮症、筋萎縮性側索硬化症などの遺伝性神経筋疾患を対象とし、患者の遺伝子に異常がある場合、その遺伝子の発現を制御することで症状改善を目指しています。研究室では、RNA結合タンパク質の異常な凝集を防ぐRNA分子設計や、スプライシング（遺伝子の読み込み過程）を制御する短鎖DNA・RNA分子の開発と改良に注力しています。これらの核酸医薬を実際に患者に届けるため、ナノメートル単位の超小型粒子や高分子を用いた運搬技術の研究も行われています。薬物が血液から筋肉や脳へ到達する際の障壁を克服し、効率的に目的の細胞へ到達させることが課題です。同時に、遺伝子検査データの解析や、核酸医薬の体内での動態追跡、そして臨床観察を通じて、治療効果と安全性の評価も進めています。これらの基礎研究と臨床実践を組み合わせることで、既存治療では対応困難な神経筋疾患の新たな治療法開発を目指しています。

※ AI（Claude）が、公開されている論文要旨から研究の問い・手法・主要な発見を事実情報として抽出・再構成して自動生成しています。誤りを含む可能性があるため、正確性は研究室公式情報でご確認ください。

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研究成果（100 件）

[2026] Rational Design of a Multivalent RNA Combining Structural Motifs Tailored to Multiple Domains of Fused in Sarcoma for Potent Inhibition of Aggregation
DOI: https://doi.org/10.1002/cbic.202500568
[2025] Iatrogenic superficial siderosis with the Pseudomeningocele after spinal surgery: a report of four cases and literature review
DOI: https://doi.org/10.1007/s00586-025-09288-9
[2025] A genome-wide association study identifies the GPM6A locus associated with age at onset in ALS
DOI: https://doi.org/10.1038/s42003-025-09168-4
[2025] Comparison of MRI-, CT- and PET-based anatomical standardization for Centiloid scale calculation in [18F]florbetapir positron emission tomography
DOI: https://doi.org/10.1007/s12149-025-02134-4
[2025] Explainable machine learning algorithm for classifying resting-state functional MRI in amyotrophic lateral sclerosis
DOI: https://doi.org/10.1016/j.neunet.2025.108359
[2025] Isoform analysis of heterozygous putative splicing variants at the allele level using nanopore long-read sequencing
DOI: https://doi.org/10.1038/s41598-025-14566-z
[2025] Enhanced Accumulation of Heteroduplex Oligonucleotides in Dystrophin-Deficient Skeletal Muscles by Single Oligonucleotide-Loaded Unit Polyion Complexes
DOI: https://doi.org/10.1021/acsomega.5c02207
[2025] Expression profiling of Epstein–Barr virus-derived microRNA in systemic chronic active EBV disease
DOI: https://doi.org/10.1007/s12185-025-04040-x
[2025] Characterization Of High-risk Plaques By Non-contrast Dual-energy CT Using Monosodium Urate Analysis: Phantom Evaluation And Clinical Translation
DOI: https://doi.org/10.1016/j.jcct.2025.05.137
[2025] Efficacy of filgrastim therapy for patients with progressive multifocal leukoencephalopathy: Two case reports
DOI: https://doi.org/10.1016/j.ijid.2025.107950

続きを表示（残り 90 件）

[2025] Audiological Progression and Treatment Outcomes in Superficial Siderosis: A 10-Year Retrospective Review of 46 Patients
DOI: https://doi.org/10.1097/mao.0000000000004535
[2025] Frequency of Guillain‐Barré Syndrome During the <scp>COVID</scp>‐19 Pandemic: A Multicenter Study
DOI: https://doi.org/10.1002/mus.28430
[2025] Refining Muscle Morphometry Through Machine Learning and Spatial Analysis
DOI: https://doi.org/10.1111/nan.70012
[2025] Specific early electroencephalogram for the diagnosis of sporadic Creutzfeldt-Jakob disease
DOI: https://doi.org/10.1080/19336896.2025.2483215
[2025] Repeated clear benefits of immunotherapy in a patient with Charcot-Marie-Tooth disease carrying a rare point mutation in PMP22
DOI: https://doi.org/10.1007/s10048-025-00808-9
[2025] G-Quadruplex-Based Splice Switching as a Therapeutic Approach in Duchenne Muscular Dystrophy
DOI: https://doi.org/10.1021/acschembio.4c00805
[2025] Pharmacokinetics and protein binding of cholesterol-conjugated heteroduplex oligonucleotide
DOI: https://doi.org/10.1016/j.jconrel.2025.02.025
[2025] An autopsy case of coexisting spinal and bulbar muscular atrophy and multiple system atrophy
DOI: https://doi.org/10.1111/neup.13031
[2025] Effect of chemical modification on the exon-skipping activity of heteroduplex oligonucleotides
DOI: https://doi.org/10.1016/j.omtn.2025.102468
[2025] Magnetomyographic evaluation of motor unit size that is robust to changes in distance to sensor
DOI: https://doi.org/10.1093/braincomms/fcaf294
[2024] DNA/RNA heteroduplex technology with cationic oligopeptide reduces class-related adverse effects of nucleic acid drugs
DOI: https://doi.org/10.1016/j.omtn.2024.102289
[2024] Characterization of Japanese multiple sclerosis patients with progression independent of relapse activity: A 2-year multicenter cohort study
DOI: https://doi.org/10.1016/j.jneuroim.2024.578407
[2024] Hydrophobicity Tuning of Cationic Polyaspartamide Derivatives for Enhanced Antisense Oligonucleotide Delivery
DOI: https://doi.org/10.1021/acs.bioconjchem.3c00456
[2024] Automated whole slide morphometry of sural nerve biopsy using machine learning
DOI: https://doi.org/10.1111/nan.12967
[2024] A Clinical Observational Study for Developing Disease Modifying Therapy Against Spinocerebellar Ataxia Type 31 (P7-3.020)
DOI: https://doi.org/10.1212/wnl.0000000000205551
[2024] CD11c <sup>high</sup> B Cell Expansion Is Associated With Severity and Brain Atrophy in Neuromyelitis Optica
DOI: https://doi.org/10.1212/nxi.0000000000200206
[2024] 5) Nucleic Acid Therapeutics for Rare Neurological Diseases
DOI: https://doi.org/10.2169/naika.113.99b
[2024] Phase II Clinical Trial of NKT Cell-targeting Glycolipid OCH-NCNP1 for Patients with Relapsing Multiple Sclerosis (P9-6.015)
DOI: https://doi.org/10.1212/wnl.0000000000204680
[2024] Conventional magnetic resonance imaging key features for distinguishing pathologically confirmed corticobasal degeneration from its mimics: a retrospective analysis of the J-VAC study
DOI: https://doi.org/10.1007/s00234-024-03432-w
[2024] Severe respiratory failure secondary to unilateral phrenic nerve injury: A case report
DOI: https://doi.org/10.1111/ncn3.12832
[2024] Effects of local reduction of endogenous α-synuclein using antisense oligonucleotides on the fibril-induced propagation of pathology through the neural network in wild-type mice
DOI: https://doi.org/10.1186/s40478-024-01766-3
[2024] Favorable efficacy and reduced acute neurotoxicity by antisense oligonucleotides with 2′,4′-BNA/LNA with 9-(aminoethoxy)phenoxazine
DOI: https://doi.org/10.1016/j.omtn.2024.102161
[2024] Eculizumab administration for myasthenia gravis also stabilizes thrombogenicity of catastrophic antiphospholipid syndrome
DOI: https://doi.org/10.1111/cen3.12790
[2024] [Peripheral Nerve and Muscle Disorders Associated with Sjögren's Syndrome].
DOI: https://doi.org/10.11477/mf.1416202648
[2024] L-arginine in patients with spinocerebellar ataxia type 6: a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial
DOI: https://doi.org/10.1016/j.eclinm.2024.102952
[2024] Functional Analysis of EBV-Derived microRNAs in Systemic Chronic Active EBV Disease Using Novel Antisense Oligonucleotides
DOI: https://doi.org/10.1182/blood-2024-206932
[2024] [Current Landscape of Tofersen in SOD-1-associated Amyotrophic Lateral Sclerosis].
DOI: https://doi.org/10.11477/mf.1416202765
[2024] Slowly expanding lesions are associated with disease activity and gray matter loss in relapse‐onset multiple sclerosis
DOI: https://doi.org/10.1111/jon.13243
[2024] Specifically Decreased Thalamic Blood Flow Following COVID-19 Infection
DOI: https://doi.org/10.1097/rlu.0000000000005478
[2024] Comparison of interaction between antimiR and microRNA versus HDO-antimiR and microRNA by molecular dynamics simulation
DOI: https://doi.org/10.1080/15257770.2024.2302526
[2024] Heteroduplex oligonucleotide technology boosts oligonucleotide splice switching activity of morpholino oligomers in a Duchenne muscular dystrophy mouse model
DOI: https://doi.org/10.1038/s41467-024-48204-5
[2024] Introduction
DOI: https://doi.org/10.2169/naika.113.1553
[2023] Peripheral administration of nanomicelle-encapsulated anti-Aβ oligomer fragment antibody reduces various toxic Aβ species in the brain
DOI: https://doi.org/10.1186/s12951-023-01772-y
[2023] Efficacy and Safety of the Natural Killer T Cell–Stimulatory Glycolipid OCH-NCNP1 for Patients With Relapsing Multiple Sclerosis: Protocol for a Randomized Placebo-Controlled Clinical Trial
DOI: https://doi.org/10.2196/46709
[2023] Genetic factors affecting survival in Japanese patients with sporadic amyotrophic lateral sclerosis: a genome-wide association study and verification in iPSC-derived motor neurons from patients
DOI: https://doi.org/10.1136/jnnp-2022-330851
[2023] Aberrant morphometric networks in Alzheimer's disease have hemispheric asymmetry and age dependence
DOI: https://doi.org/10.1111/ejn.16225
[2023] Serial changes in regional cerebral blood flow in Gerstmann–Sträussler–Scheinker disease caused by a Pro-to-Leu mutation at codon 105 in the prion protein gene
DOI: https://doi.org/10.1080/19336896.2023.2256928
[2023] Amplified Risk of Intracranial Artery Stenosis/Occlusion Associated With RNF213 p.R4810K in Familial Hypercholesterolemia
DOI: https://doi.org/10.1016/j.jacasi.2023.03.011
[2023] Verification of propagation hypothesis in patients with sporadic hand onset amyotrophic lateral sclerosis
DOI: https://doi.org/10.1007/s13760-023-02297-9
[2023] Altered brain energy metabolism related to astrocytes in tauopathies
DOI: https://doi.org/10.1002/alz.059834
[2023] Substitution of Glu to Lys at Codon 332 on the <i>GFAP</i> Gene Alone Is Causative for Adult-onset Alexander Disease
DOI: https://doi.org/10.2169/internalmedicine.1726-23
[2023] <scp>TDP</scp>‐43 N‐terminal domain dimerisation or spatial separation by <scp>RNA</scp> binding decreases its propensity to aggregate
DOI: https://doi.org/10.1002/1873-3468.14635
[2023] Temporal Relationship between Impairment of Cerebellar Motor Learning and Deterioration of Ataxia in Patients with Cerebellar Degeneration
DOI: https://doi.org/10.1007/s12311-023-01545-1
[2023] Angubindin-1 opens the blood–brain barrier in vivo for delivery of antisense oligonucleotide to the central nervous system (S14.009)
DOI: https://doi.org/10.1212/wnl.0000000000204102
[2023] Teaching NeuroImages: Parkinsonism Secondary to a Metastatic Lesion Involving the Substantia Nigra
DOI: https://doi.org/10.1212/wnl.0000000000207019
[2023] High-dose ubiquinol supplementation in multiple-system atrophy: a multicentre, randomised, double-blinded, placebo-controlled phase 2 trial
DOI: https://doi.org/10.1016/j.eclinm.2023.101920
[2023] Effects of combinations of gapmer antisense oligonucleotides on the target reduction
DOI: https://doi.org/10.1007/s11033-022-08224-0
[2023] Circulating Extracellular Vesicle-Propagated microRNA Signature as a Vascular Calcification Factor in Chronic Kidney Disease
DOI: https://doi.org/10.1161/circresaha.122.321939
[2023] Receptor mediated tissue targeted delivery of oligonucleotide
DOI: https://doi.org/10.2745/dds.38.8
[2023] Preferential delivery of lipid-ligand conjugated DNA/RNA heteroduplex oligonucleotide to ischemic brain in hyperacute stage
DOI: https://doi.org/10.1016/j.ymthe.2023.01.016
[2023] Correlation of callosal angle at the splenium with gait and cognition in normal pressure hydrocephalus
DOI: https://doi.org/10.3171/2022.12.jns221825
[2023] Clinical course of pathologically confirmed corticobasal degeneration and corticobasal syndrome
DOI: https://doi.org/10.1093/braincomms/fcad296
[2022] Spinocerebellar Ataxia Type 31 Exacerbated by Anti-amino Terminal of Alpha-enolase Autoantibodies
DOI: https://doi.org/10.2169/internalmedicine.8883-21
[2022] Prediction of tissue-of-origin of early stage cancers using serum miRNomes
DOI: https://doi.org/10.1093/jncics/pkac080
[2022] Turning and multitask gait unmask gait disturbance in mild‐to‐moderate multiple sclerosis: Underlying specific cortical thinning and connecting fibers damage
DOI: https://doi.org/10.1002/hbm.26151
[2022] Expression Profile of EBV-Derived Micro-RNA in Systemic Chronic Active EBV Disease
DOI: https://doi.org/10.1182/blood-2022-169693
[2022] Quantitative Model Analysis and Simulation of Pharmacokinetics and Metastasis-Associated Lung Adenocarcinoma 1 RNA Knockdown Effect After Systemic Administration of Cholesterol-Conjugated DNA/RNA Heteroduplex Oligonucleotide Crossing Blood–Brain Barrier of Mice
DOI: https://doi.org/10.1124/jpet.122.001331
[2022] Clinical impact of amyloid PET using 18F-florbetapir in patients with cognitive impairment and suspected Alzheimer’s disease: a multicenter study
DOI: https://doi.org/10.1007/s12149-022-01792-y
[2022] Case Report: Dural Dissection With Ventral Spinal Fluid-Filled Collection in Superficial Siderosis: Insights Into the Pathology From Anterior-Approached Surgical Cases
DOI: https://doi.org/10.3389/fneur.2022.919280
[2022] Paraneoplastic Cerebellar Degeneration With P/Q-VGCC vs Yo Autoantibodies
DOI: https://doi.org/10.1212/nxi.0000000000200006
[2022] Systemic DNA/RNA heteroduplex oligonucleotide administration for regulating the gene expression of dorsal root ganglion and sciatic nerve
DOI: https://doi.org/10.1016/j.omtn.2022.05.006
[2022] Neuronal internalization of immunoglobulin G injected into the mouse brain by a novel absorption strategy to avoid unwanted interaction with immune complex using centrifugal filtration
DOI: https://doi.org/10.1007/s00418-022-02107-y
[2022] Specific electroencephalogram features in the very early phases of sporadic Creutzfeldt–Jakob disease
DOI: https://doi.org/10.1016/j.jns.2022.120265
[2022] Parallel gold enhancement of quantum dots 565/655 for double-labelling correlative light and electron microscopy on human autopsied samples
DOI: https://doi.org/10.1038/s41598-022-09849-8
[2022] The Role of Long Noncoding RNA MALAT1 in Diabetic Polyneuropathy and the Impact of Its Silencing in the Dorsal Root Ganglion by a DNA/RNA Heteroduplex Oligonucleotide
DOI: https://doi.org/10.2337/db21-0918
[2022] Amyloid-β oligomers interact with NMDA receptors containing GluN2B subunits and metabotropic glutamate receptor 1 in primary cortical neurons: Relevance to the synapse pathology of Alzheimer’s disease
DOI: https://doi.org/10.1016/j.neures.2022.03.001
[2022] Regulation of activated microglia and macrophages by systemically administered DNA/RNA heteroduplex oligonucleotides
DOI: https://doi.org/10.1016/j.ymthe.2022.02.019
[2022] High-efficacy therapy reduces subcortical grey matter volume loss in Japanese patients with relapse-onset multiple sclerosis: A 2-year cohort study
DOI: https://doi.org/10.1016/j.msard.2022.104077
[2022] Serial ultrathin sections to identify ultrastructural localization of GLUT1 molecules in vesicles in brain endothelial cells—correlative light and electron microscopy in depth
DOI: https://doi.org/10.1093/jmicro/dfac005
[2022] Thymidine Kinase 2 and Mitochondrial Protein COX I in the Cerebellum of Patients with Spinocerebellar Ataxia Type 31 Caused by Penta-nucleotide Repeats (TTCCA)n
DOI: https://doi.org/10.1007/s12311-021-01364-2
[2022] [Current Status of University-Originated Startups and Careers for Its Establishment].
DOI: https://doi.org/10.11477/mf.1416201983
[2022] Change of intracellular calcium level causes acute neurotoxicity by antisense oligonucleotides via CSF route
DOI: https://doi.org/10.1016/j.omtn.2022.12.010
[2021] Reversible Reduction of Dopamine Transporter Uptake in a Case of Hydrocephalus
DOI: https://doi.org/10.1002/ana.26130
[2021] Short DNA/RNA heteroduplex oligonucleotide interacting proteins are key regulators of target gene silencing
DOI: https://doi.org/10.1093/nar/gkab258
[2021] Evaluation of the risk factors predicting thrombotic complications associated with intravenous immunoglobulin therapy in neuroimmunological diseases
DOI: https://doi.org/10.1007/s10072-021-05192-4
[2021] Oculo-dento-digital Dysplasia Presenting as Spastic Paraparesis Which Was Successfully Treated by Intrathecal Baclofen Therapy
DOI: https://doi.org/10.2169/internalmedicine.6145-20
[2021] HLA genotype-clinical phenotype correlations in multiple sclerosis and neuromyelitis optica spectrum disorders based on Japan MS/NMOSD Biobank data
DOI: https://doi.org/10.1038/s41598-020-79833-7
[2021] We Should Pay Attention to "Referred Pain" - A Case of Acute Myocardial Infarction That Masked and Delayed the Diagnosis of Esophageal Perforation
DOI: https://doi.org/10.2169/internalmedicine.7694-21
[2021] Cholesterol-functionalized DNA/RNA heteroduplexes cross the blood–brain barrier and knock down genes in the rodent CNS
DOI: https://doi.org/10.1038/s41587-021-00972-x
[2021] A patient with generalized myasthenia gravis facing an impending crisis triggered by early fast‐acting treatment
DOI: https://doi.org/10.1111/cen3.12681
[2021] Tap-Elicited Clonus of the Biceps and Triceps Brachii
DOI: https://doi.org/10.1001/jamaneurol.2021.4273
[2021] Mutation screening of the DNAJC7 gene in Japanese patients with sporadic amyotrophic lateral sclerosis
DOI: https://doi.org/10.1016/j.neurobiolaging.2021.12.002
[2021] DNA/RNA heteroduplex oligonucleotide technology for regulating lymphocytes in vivo
DOI: https://doi.org/10.1038/s41467-021-26902-8
[2021] Extracellular vesicles derived from mesenchymal stromal cells mediate endogenous cell growth and migration via the CXCL5 and CXCL6/CXCR2 axes and repair menisci
DOI: https://doi.org/10.1186/s13287-021-02481-9
[2021] Effective silencing of miR-126 after ischemic stroke by means of intravenous α-tocopherol–conjugated heteroduplex oligonucleotide in mice
DOI: https://doi.org/10.1038/s41598-021-93666-y
[2021] A Japanese Multicenter Study on PET and Other Biomarkers for Subjects with Potential Preclinical and Prodromal Alzheimer's Disease
DOI: https://doi.org/10.14283/jpad.2021.37
[2021] DNA damage in embryonic neural stem cell determines FTLDs’ fate via early-stage neuronal necrosis
DOI: https://doi.org/10.26508/lsa.202101022
[2021] Neuropathology of SCA34 showing widespread oligodendroglial pathology with vacuolar white matter degeneration: a case study
DOI: https://doi.org/10.1186/s40478-021-01272-w
[2021] Sacral dural arteriovenous fistula mimicking multiple mononeuropathy
DOI: https://doi.org/10.1016/j.clineuro.2021.106993
[2021] Quantitative clinical and radiological recovery in post-operative patients with superficial siderosis by an iron chelator
DOI: https://doi.org/10.1007/s00415-021-10844-8

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AI 要約（直近 5 年の研究成果）

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研究成果（100 件）

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