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Elucidation of pathomecanism for Fukuyama muscular dystrophy and dystroglycanopathy and their drug developmen

Research Project

Project/Area Number 26253057
Research Category

Grant-in-Aid for Scientific Research (A)

Allocation TypeSingle-year Grants
Section一般
Research Field Neurology
Research InstitutionKobe University

Principal Investigator

Toda Tatsusi  神戸大学, 医学研究科, 教授 (30262025)

Project Period (FY) 2014-04-01 – 2017-03-31
Project Status Completed (Fiscal Year 2017)
Budget Amount *help
¥41,470,000 (Direct Cost: ¥31,900,000、Indirect Cost: ¥9,570,000)
Fiscal Year 2016: ¥11,960,000 (Direct Cost: ¥9,200,000、Indirect Cost: ¥2,760,000)
Fiscal Year 2015: ¥13,520,000 (Direct Cost: ¥10,400,000、Indirect Cost: ¥3,120,000)
Fiscal Year 2014: ¥15,990,000 (Direct Cost: ¥12,300,000、Indirect Cost: ¥3,690,000)
Keywords福山型筋ジストロフィー / ジストログリパノカチー / リビトールリン酸 / フクチン / アンチセンス治療 / ジストログリカノパチー / 臨床神経分子遺伝学 / レトロトランスポゾン / LARGE
Outline of Final Research Achievements

We re-designed AONs precisely around the splice sites and assessed the efficacy for exon trap inhibition of these AONs in Fukuyama CMD patient cells and model mice. We finally selected one best candidate AON. We demonstrated that fukutin is required for LARGE-dependent rescue of α-DG glycosylation. We further identified the previously unknown glycan unit ribitol 5-phosphate (Rbo5P), a phosphoric ester of pentose alcohol, as a tandem repeat that functions as a scaffold for the formation of the ligand-binding moiety of α-DG. We determined the enzyme activities of three major α-DGpathy-causing proteins to be involved in the synthesis of tandem Rbo5P. Isoprenoid synthase domain-containing (ISPD) is cytidine diphosphate ribitol (CDP-Rbo) synthase. Fukutin and fukutin-related protein are Rbo5P transferases that use CDP-Rbo. We also indicate that spatiotemporal persistence of functionally glycosylated dystroglycan may be crucial for brain development.

Report

(4 results)
  • 2017 Final Research Report ( PDF )
  • 2016 Annual Research Report
  • 2015 Annual Research Report
  • 2014 Annual Research Report
  • Research Products

    (62 results)

All 2018 2017 2016 2015 2014 Other

All Journal Article (27 results) (of which Int'l Joint Research: 2 results,  Peer Reviewed: 25 results,  Open Access: 25 results,  Acknowledgement Compliant: 5 results) Presentation (31 results) (of which Int'l Joint Research: 14 results,  Invited: 28 results) Remarks (1 results) Patent(Industrial Property Rights) (3 results)

  • [Journal Article] Cell endogenous activities of fukutin and FKRP coexist with the ribitol xylosyltransferase, TMEM5.2018

    • Author(s)
      Nishihara, R., Kobayashi, K., Imae, R., Tsumoto, H., Manya, H., Mizuno, M., Kanagawa, M., Endo, T., and Toda, T.
    • Journal Title

      Biochemical and Biophysical Research Communications

      Volume: 497 Issue: 4 Pages: 1025-1030

    • DOI

      10.1016/j.bbrc.2018.02.162

    • Related Report
      2016 Annual Research Report
    • Peer Reviewed / Open Access
  • [Journal Article] The Ror1 receptor tyrosine kinase plays a critical role in regulating satellite cell proliferation during regeneration of injured muscle2017

    • Author(s)
      Kamizaki Koki、Doi Ryosuke、Hayashi Makoto、Saji Takeshi、Kanagawa Motoi、Toda Tatsushi、Fukada So-ichiro、Ho Hsin-Yi Henry、Greenberg Michael Eldon、Endo Mitsuharu、Minami Yasuhiro
    • Journal Title

      Journal of Biological Chemistry

      Volume: 292 Issue: 38 Pages: 15939-15951

    • DOI

      10.1074/jbc.m117.785709

    • Related Report
      2016 Annual Research Report
    • Peer Reviewed / Open Access / Int'l Joint Research
  • [Journal Article] Cardiac involvement in Fukuyama muscular dystrophy is less severe than in Duchenne muscular dystrophy.2017

    • Author(s)
      Yamamoto T, Taniguchi-Ikeda M, Awano H, Matsumoto M, Lee T, Harada R, Imanishi T, Hayashi N, Sakai Y, Morioka I, Takeshima Y, Iijima K, Saegusa J, Toda T.
    • Journal Title

      Brain and Development

      Volume: 39 Issue: 10 Pages: 861-868

    • DOI

      10.1016/j.braindev.2017.05.008

    • Related Report
      2016 Annual Research Report
    • Peer Reviewed / Open Access
  • [Journal Article] Deep-intronic variant of fukutin is the most prevalent point mutation of Fukuyama congenital muscular dystrophy in Japan2017

    • Author(s)
      Kobayashi Kazuhiro、Kato Reiko、Kondo-Iida Eri、Taniguchi-Ikeda Mariko、Osawa Makiko、Saito Kayoko、Toda Tatsushi
    • Journal Title

      Journal of Human Genetics

      Volume: 62 Issue: 11 Pages: 945-948

    • DOI

      10.1038/jhg.2017.71

    • Related Report
      2016 Annual Research Report
    • Peer Reviewed / Open Access
  • [Journal Article] Novel FKRP mutations in a Japanese MDC1C sibship clinically diagnosed with Fukuyama congenital muscular dystrophy2017

    • Author(s)
      Yoshioka Mieko、Kobayashi Kazuhiro、Toda Tatsushi
    • Journal Title

      Brain and Development

      Volume: 39 Issue: 10 Pages: 869-872

    • DOI

      10.1016/j.braindev.2017.05.013

    • Related Report
      2016 Annual Research Report
    • Peer Reviewed / Open Access
  • [Journal Article] Muscular Dystrophy with Ribitol-Phosphate Deficiency: A Novel Post-Translational Mechanism in Dystroglycanopathy2017

    • Author(s)
      Kanagawa Motoi、Toda Tatsushi
    • Journal Title

      Journal of Neuromuscular Diseases

      Volume: 4 Issue: 4 Pages: 259-267

    • DOI

      10.3233/jnd-170255

    • NAID

      120006811130

    • Related Report
      2016 Annual Research Report
    • Peer Reviewed / Open Access
  • [Journal Article] Analysis of phenotype, enzyme activity, and genotype of Chinese patients with POMT1 mutation2016

    • Author(s)
      Haipo Yang, Hiroshi Manya, Kazuhiro Kobayashi, HUI JIAO, Xiaona Fu, Jiangxi Xiao, Xiaoqing Li, Jingmin wang, Yuwu Jiang, TATSUSHI TODA, Tamao Endo, Xiru Wu,Hui Xiong,
    • Journal Title

      J. Hum. Genet

      Volume: 印刷中 Issue: 8 Pages: 753-759

    • DOI

      10.1038/jhg.2016.42

    • NAID

      40020923851

    • Related Report
      2016 Annual Research Report
    • Peer Reviewed / Open Access / Int'l Joint Research / Acknowledgement Compliant
  • [Journal Article] Carbohydrate-binding domain of the POMGnT1 stem region modulates O-mannosylation sites of α-dystroglycan.2016

    • Author(s)
      Kuwabara N, Manya H, Yamada T, Tateno H, Kanagawa M, Kobayashi K, Akasaka-Manya K, Hirose Y, Mizuno M, Ikeguchi M, Toda T, Hirabayashi J, Senda T, Endo T, Kato R.
    • Journal Title

      Proc Natl Acad Sci U S A.

      Volume: 113 Issue: 33 Pages: 9280-9285

    • DOI

      10.1073/pnas.1525545113

    • Related Report
      2016 Annual Research Report 2015 Annual Research Report
    • Peer Reviewed / Open Access
  • [Journal Article] Mechanistic aspects of the formation of α-dystroglycan and therapeutic research for the treatment of α-dystroglycanopathy: A review.2016

    • Author(s)
      Taniguchi-Ikeda M, Morioka I, Iijima K, Toda T.
    • Journal Title

      Mol Aspects Med.

      Volume: 51 Pages: 115-124

    • DOI

      10.1016/j.mam.2016.07.003

    • Related Report
      2016 Annual Research Report
    • Peer Reviewed / Open Access / Acknowledgement Compliant
  • [Journal Article] The muscular dystrophy gene TMEM5encodes a ribitol β1-4 Xylosyltransferase required for the functional glycosylation of dystroglycan.2016

    • Author(s)
      Manya, H., Yamaguchi, Y., Kanagawa, M., Kobayashi, K., Tajiri, M., Akasaka-Manya, K., Kawakami, H., Mizuno, M., Wada, Y., Toda, T., and Endo, T.
    • Journal Title

      J. Biol. Chem

      Volume: 291 Issue: 47 Pages: 24618-24627

    • DOI

      10.1074/jbc.m116.751917

    • Related Report
      2016 Annual Research Report
    • Peer Reviewed / Open Access / Acknowledgement Compliant
  • [Journal Article] Next-generation sequencing discloses a nonsense mutation in the dystrophin gene from long preserved dried umbilical cord and low-level somatic mosaicism in the proband mother.2016

    • Author(s)
      Taniguchi-Ikeda M, Takeshima Y, Lee T, Nishiyama M, Awano H, Yagi M, Unzaki A, Nozu K, Nishio H, Matsuo M, Kurahashi H, Toda T, Morioka I, Iijima K.
    • Journal Title

      J Hum Genet.

      Volume: 61 Issue: 4 Pages: 351-355

    • DOI

      10.1038/jhg.2015.157

    • NAID

      40020802923

    • Related Report
      2015 Annual Research Report
    • Peer Reviewed / Open Access
  • [Journal Article] Identification of a post-translational modification with ribitol-phosphate and its defect in muscular dystrophy2016

    • Author(s)
      Kanagawa M, Kobayashi K, Tajiri M, Manya H, Kuga A, Yamaguchi Y, Akasaka-Manya K, Furukawa J, Mizuno M, Kawakami H, Shinohara Y, Wada Y, Endo T, Toda T.
    • Journal Title

      Cell Reports

      Volume: 14 Issue: 9 Pages: 2209-2223

    • DOI

      10.1016/j.celrep.2016.02.017

    • NAID

      120005728438

    • Related Report
      2015 Annual Research Report
    • Peer Reviewed / Open Access / Acknowledgement Compliant
  • [Journal Article] A de novo mutation of the MYH7 gene in a large Chinese family with autosomal dominant myopathy2015

    • Author(s)
      Oda T, Xiong H, Kobayashi K, Wang S, Satake W, Jiao H, Yang Y, Cha PC, Hayashi Y, Nishino I, Suzuki Y, Sugano S, Wu X, Toda T.
    • Journal Title

      Human Genome Variation

      Volume: 2 Issue: 1 Pages: 15022-15022

    • DOI

      10.1038/hgv.2015.22

    • Related Report
      2015 Annual Research Report
    • Peer Reviewed / Open Access
  • [Journal Article] A novel PIGN mutation and prenatal diagnosis of inherited glycosylphosphatidylinositol deficiency.2015

    • Author(s)
      Nakagawa T, Taniguchi-Ikeda M, Murakami Y, Nakamura S, Motooka D, Emoto T, Satake W, Nishiyama M, Toyoshima D, Morisada N, Takada S, Tairaku S, Okamoto N, Morioka I, Kurahashi H, Toda T, Kinoshita T, Iijima K.
    • Journal Title

      American Journal of Medical Genetics PartA

      Volume: 170 Issue: 1 Pages: 183-188

    • DOI

      10.1002/ajmg.a.37397

    • Related Report
      2015 Annual Research Report
    • Peer Reviewed / Open Access / Acknowledgement Compliant
  • [Journal Article] ABCG2 variant has opposing effects on onset ages of Parkinson's disease and gout.2015

    • Author(s)
      Matsuo H, Tomiyama H, Satake W, Chiba T, Onoue H, Kawamura Y, Nakayama A, ShimizuS, Sakiyama M, Funayama M, Nishioka K, Shimizu T, Kaida K, Kamakura K, Toda T, Hattori N, ShinomiyaN.
    • Journal Title

      Ann Clin Transl Neurol

      Volume: 2 Issue: 3 Pages: 302-306

    • DOI

      10.1002/acn3.167

    • Related Report
      2014 Annual Research Report
    • Peer Reviewed / Open Access
  • [Journal Article] Fukutin is prerequisite to ameliorate muscular dystrophic phenotype by myofiber-selective LARGE expression.2015

    • Author(s)
      Ohtsuka Y, Kanagawa M, Yu CC, Ito C, Chiyo T, Kobayashi K, Okada T, Takeda S’I, Toda T.
    • Journal Title

      Sci Rep

      Volume: 5 Issue: 1 Pages: 8316-8316

    • DOI

      10.1038/srep08316

    • NAID

      120005600773

    • Related Report
      2014 Annual Research Report
    • Peer Reviewed / Open Access
  • [Journal Article] CHCHD2 mutations in autosomal dominant late-onset Parkinson’s disease: a genome-wide linkage and sequencing study.2015

    • Author(s)
      Funayama M, Ohe K, Amo T, Furuya N, Yamaguchi J, Saiki S, Yuanzhe L, Ogaki K, Ando M, Yoshinon H, Tomiyama H, Nishioka K, Hasegawa K, Saiki H, Satake W, Mogushi K, Sasaki R, Kokubo Y, Kuzuhara S, Toda T, Mizuno Y, Uchiyama Y, Ohno K, Hattori N.
    • Journal Title

      Lancet Neurol

      Volume: 14 Issue: 3 Pages: 274-282

    • DOI

      10.1016/s1474-4422(14)70266-2

    • Related Report
      2014 Annual Research Report
    • Peer Reviewed / Open Access
  • [Journal Article] TRPV2 is critical for the maintenance of cardiac structure and function in mice.2014

    • Author(s)
      Katanosaka Y, Iwasaki K, Ujihara Y, Takatsu S, Nishitsuji K, Kanagawa M, Sudo A, Toda T, Katanosaka K, Mohri S, Naruse K.
    • Journal Title

      Nat Comm

      Volume: 5 Issue: 1 Pages: 3932-3932

    • DOI

      10.1038/ncomms4932

    • NAID

      120005690060

    • Related Report
      2014 Annual Research Report
    • Peer Reviewed / Open Access
  • [Journal Article] Peripheral leukocyte anomaly detected with routine automated hematology analyzer sensitive to adipose triglyceride lipase deficiency manifesting neutral lipid storage disease with myopathy/triglyceride deposit cardiomyovasculopathy.2014

    • Author(s)
      Suzuki A, Nagasaka H, Ochi Y, Kobayashi K, Nakamura H, Nakatani D, S Yamaguchi, Yamaki S, Wada A, Shirata Y, Hui SP, Toda T, Kuroda H, Chiba H, Hirano K.
    • Journal Title

      Mol Genet Metab Rep

      Volume: 1 Pages: 249-253

    • DOI

      10.1016/j.ymgmr.2014.05.001

    • Related Report
      2014 Annual Research Report
    • Peer Reviewed / Open Access
  • [Journal Article] Disease-associated marked hyperalphalipoproteinemia.2014

    • Author(s)
      Hirano K, Nagasaka H, Kobayashi K, Yamaguchi S, Suzuki A, Toda T, Doyu M.
    • Journal Title

      Mol Genet Metab Rep

      Volume: 1 Pages: 264-268

    • DOI

      10.1016/j.ymgmr.2014.06.001

    • Related Report
      2014 Annual Research Report
    • Peer Reviewed / Open Access
  • [Journal Article] The role of Pak-interacting exchange factor-β phosphorylation at serines 340 and 583 by PKCγ in dopamine release.2014

    • Author(s)
      Shirafuji T, Ueyama T, Yoshino K, Takahashi H, Adachi N, Ago Y, Koda K, Nashida T, Hiramatsu N, Matsuda T, Toda T, Sakai N, Saito N.
    • Journal Title

      J Neurosci

      Volume: 34 Issue: 28 Pages: 9268-9280

    • DOI

      10.1523/jneurosci.4278-13.2014

    • NAID

      120005619641

    • Related Report
      2014 Annual Research Report
    • Peer Reviewed / Open Access
  • [Journal Article] Association of the ASCO classification with the executive function subscores of the Montreal Cognitive Assessment in patients with post-ischemic stroke.2014

    • Author(s)
      Washida K, IharaM, Tachibana H, Sekiguchi K, Kowa H, Kanda F, Toda T.
    • Journal Title

      J Stroke Cerebrovasc Dis

      Volume: 23 Issue: 9 Pages: 2250-2255

    • DOI

      10.1016/j.jstrokecerebrovasdis.2014.04.009

    • Related Report
      2014 Annual Research Report
    • Peer Reviewed / Open Access
  • [Journal Article] Overexpression of LARGE suppresses muscle regeneration via down-regulation of insulin-like growth factor 1 and aggravates muscular dystrophy in mice2014

    • Author(s)
      Saito F, Kanagawa M, Ikeda M, Hagiwara H, Masaki T, Ohkuma H, Katanosaka Y, Shimizu T, Sonoo M, Toda T, Matsumura K.
    • Journal Title

      Hum Mol Genet

      Volume: 23 Issue: 17 Pages: 4543

    • DOI

      10.1093/hmg/ddu168

    • URL

      https://pure.teikyo.jp/en/publications/3a900055-4ad6-4411-946d-7f072c2d44ee

    • Related Report
      2014 Annual Research Report
    • Peer Reviewed / Open Access
  • [Journal Article] Contribution of dysferlin deficiency to skeletal muscle pathology in asymptomatic and severe dystroglycanopathy models: generation of a new model for fukuyama congenital muscular dystrophy.2014

    • Author(s)
      Kanagawa M, Lu Z, Ito C, Matsuda C, Miyake K, Toda T.
    • Journal Title

      Plos ONE

      Volume: 9 Issue: 9 Pages: e106721-e106721

    • DOI

      10.1371/journal.pone.0106721

    • Related Report
      2014 Annual Research Report
    • Peer Reviewed / Open Access
  • [Journal Article] Japanese founder duplications/triplications involving BHLHA9 are associated with split-hand/foot malformation with or without long bone deficiency and Gollop-Wolfgang complex.2014

    • Author(s)
      Nagata E, Kano H, Kato F, Yamaguchi R, Nakashima S, Takayama S, Kosaki R, Tonoki H, Mizuno S, Watanabe S, Yoshiura KI, Kosho T, Hasegawa T, Kimizuka M, Suzuki A, Shimizu K, Ohashi H, Haga N, Numabe H, Horii E, Nagai T, Yoshihashi H, Nishimura G, Toda T, et al.
    • Journal Title

      Orphanet J Rare Dis

      Volume: 9 Issue: 1 Pages: 125-125

    • DOI

      10.1186/s13023-014-0125-5

    • Related Report
      2014 Annual Research Report
    • Peer Reviewed / Open Access
  • [Journal Article] 医学と医療の最前線 筋ジストロフィー治療の新しい展開2014

    • Author(s)
      戸田達史
    • Journal Title

      日本内科学会雑誌

      Volume: 103 Pages: 2820-2828

    • Related Report
      2014 Annual Research Report
  • [Journal Article] 福山型筋ジストロフィー 仕組みの解明 治療法開発2014

    • Author(s)
      戸田達史
    • Journal Title

      関西実験動物研究会

      Volume: 会報36号 Pages: 83-83

    • Related Report
      2014 Annual Research Report
  • [Presentation] Identification of a new glycosylation with ribitol-phosphate and its defect in ISPD, FCMD and LGMD2I2017

    • Author(s)
      Tatsushi Toda
    • Organizer
      5th International Workshop for Glycosylation Defects in Muscular Dystrophies
    • Related Report
      2016 Annual Research Report
    • Int'l Joint Research / Invited
  • [Presentation] Fukutin is Ribitol 5-Phosphate Transferase: Recent Advance in Fukuyama Muscular Dystrophy2017

    • Author(s)
      Tatsushi Toda
    • Organizer
      14th Asian and Oceanian Congress of Child Neurology
    • Related Report
      2016 Annual Research Report
    • Int'l Joint Research / Invited
  • [Presentation] Genetics and nucleic acid therapeutics of Fukuyama muscular dystrophy2017

    • Author(s)
      Tatsushi Toda
    • Organizer
      第一回国際がんプレシジョン医療カンファレンス
    • Related Report
      2016 Annual Research Report
    • Int'l Joint Research / Invited
  • [Presentation] 福山型筋ジストロフィーを含めた糖鎖合成異常症の系統的な解明と新しい糖鎖の発見2017

    • Author(s)
      戸田達史
    • Organizer
      日本筋学会第3回学術集会
    • Related Report
      2016 Annual Research Report
    • Invited
  • [Presentation] Genomewide Analysis and Molecular Targeting for Neurological Diseases2017

    • Author(s)
      Tatsushi Toda
    • Organizer
      International Symposium on Smart Brain Medical Informatics and Engineering
    • Related Report
      2016 Annual Research Report
    • Int'l Joint Research / Invited
  • [Presentation] Identification of a posttranslational modification with ribitol-phosphate and its defect in muscular dystrophy2017

    • Author(s)
      Tatsushi Toda
    • Organizer
      East Asian Unit of Himan Genetics Society
    • Related Report
      2016 Annual Research Report
    • Int'l Joint Research / Invited
  • [Presentation] 疾患遺伝子の発見から新しい治療法の開発2017

    • Author(s)
      戸田達史
    • Organizer
      生命科学系合同年次大会シンポジウム
    • Related Report
      2016 Annual Research Report
    • Invited
  • [Presentation] 福山型筋ジストロフィーをはじめとするジストログリカン異常症の分子病態と治療2016

    • Author(s)
      戸田 達史
    • Organizer
      第2回日本筋学会学術集会
    • Place of Presentation
      国立精神・神経医療研究センター(東京)
    • Year and Date
      2016-08-06
    • Related Report
      2015 Annual Research Report
    • Invited
  • [Presentation] 福山型先天性筋ジストロフィーの治療法開発2016

    • Author(s)
      戸田 達史
    • Organizer
      第57回日本神経学会学術大会
    • Place of Presentation
      神戸国際会議場・神戸国際展示場・神戸ポートピアホテル(神戸)
    • Year and Date
      2016-05-19
    • Related Report
      2015 Annual Research Report
    • Invited
  • [Presentation] Gene Therapy for Transposon Disease2016

    • Author(s)
      戸田 達史
    • Organizer
      第57回日本神経学会学術大会
    • Place of Presentation
      神戸国際会議場・神戸国際展示場・神戸ポートピアホテル(神戸)
    • Year and Date
      2016-05-19
    • Related Report
      2015 Annual Research Report
    • Invited
  • [Presentation] 筋ジストロフィーの分子機構と治療戦略2016

    • Author(s)
      戸田 達史
    • Organizer
      第113回日本内科学会
    • Place of Presentation
      東京国際フォーラム(東京)
    • Year and Date
      2016-04-15
    • Related Report
      2015 Annual Research Report
    • Invited
  • [Presentation] 筋ジストロフィーの分子機構と治療戦略2016

    • Author(s)
      戸田 達史
    • Organizer
      第113回日本内科学会
    • Related Report
      2016 Annual Research Report
    • Invited
  • [Presentation] 福山型先天性筋ジストロフィーの治療法開発2016

    • Author(s)
      戸田 達史
    • Organizer
      第57回日本神経学会学術大会
    • Related Report
      2016 Annual Research Report
    • Invited
  • [Presentation] Gene Therapy for Transposon Disease2016

    • Author(s)
      Tatsushi Toda
    • Organizer
      第57回日本神経学会学術大会
    • Related Report
      2016 Annual Research Report
    • Invited
  • [Presentation] 福山型筋ジストロフィーをはじめとするジストログリカン異常症の分子病態と治療2016

    • Author(s)
      戸田 達史
    • Organizer
      第2回日本筋学会学術集会
    • Related Report
      2016 Annual Research Report
    • Invited
  • [Presentation] Identification of a post-translational modification with ribitol-phosphate and its defect in muscular dystrophy.2016

    • Author(s)
      Tatsushi Toda
    • Organizer
      Warren Workshop VI 2016
    • Related Report
      2016 Annual Research Report 2015 Annual Research Report
    • Int'l Joint Research / Invited
  • [Presentation] Identification of a Post-translational Modification with Ribitol-Phosphate and Its Defect in Muscular Dystrophy.2016

    • Author(s)
      Tatsushi Toda
    • Organizer
      21st WMS congress 2016
    • Related Report
      2016 Annual Research Report
    • Int'l Joint Research
  • [Presentation] Genome-wide Analysis and Molecular Targeting Therapy for Neuro-Muscular deisease.2016

    • Author(s)
      Tatsushi Toda
    • Organizer
      2016 Taiwan-Japan Joint Conference on Genomic Studies and Annual Retreat of Taiwan Genomics and Genetics Society.
    • Related Report
      2016 Annual Research Report
    • Int'l Joint Research / Invited
  • [Presentation] 福山型筋ジストロフィー及び類縁疾患の糖鎖病態と分子標的治療2015

    • Author(s)
      戸田 達史
    • Organizer
      第38回日本分子生物学会年会、第88回日本生化学会大会 合同大会
    • Place of Presentation
      神戸ポートアイランド(神戸)
    • Year and Date
      2015-12-02
    • Related Report
      2015 Annual Research Report
    • Invited
  • [Presentation] Recent Advances in Muscular Dystrophy2015

    • Author(s)
      Tatsushi Toda
    • Organizer
      UNIVERSITY OF WASHINGTON-KOBE UNIVERSITY SYMPOSIUM ON CELL SIGNALING
    • Place of Presentation
      seattle, the U.S.A.
    • Year and Date
      2015-09-10
    • Related Report
      2015 Annual Research Report
    • Int'l Joint Research / Invited
  • [Presentation] Molecular pathogenesis & molecular targeting therapy for a- dystroglycanopathy.2015

    • Author(s)
      Tatsushi Toda
    • Organizer
      The 10th Japanese-French Workshop “New advances in treatments of neuromuscular diseases: From Basic to Applied Myology.”
    • Place of Presentation
      Paris, France
    • Year and Date
      2015-07-03
    • Related Report
      2015 Annual Research Report
    • Int'l Joint Research / Invited
  • [Presentation] Use of antisense oligonucleotides in FCMD mouse models.2015

    • Author(s)
      Tatsushi Toda
    • Organizer
      212th ENMC International Workshop Animal models of Congenital Muscular Dystrophies
    • Place of Presentation
      Naarden, Netherlands
    • Year and Date
      2015-05-19
    • Related Report
      2015 Annual Research Report
    • Int'l Joint Research / Invited
  • [Presentation] Genomewide analysis and molecular targeting therapy for neurological diseases2015

    • Author(s)
      Tatsushi Toda
    • Organizer
      nature conference - Genomic Variations in Precision Medicine 2015
    • Place of Presentation
      Changsha, China
    • Year and Date
      2015-05-17
    • Related Report
      2015 Annual Research Report
    • Int'l Joint Research / Invited
  • [Presentation] Recent Advances in Genetics of α-Dystroglycanopathy.2015

    • Author(s)
      Tatsushi Toda
    • Organizer
      the 13th Asian & Oceanian Congress of Child Neurology (AOCCN)
    • Place of Presentation
      Taipei, Taiwan
    • Year and Date
      2015-05-14
    • Related Report
      2015 Annual Research Report
    • Int'l Joint Research / Invited
  • [Presentation] α-dystroglycanopathy and molecular targeting therapy.2015

    • Author(s)
      Tatsushi Toda
    • Organizer
      Fourth International Workshop for Glycosylation Defects in Muscular Dystrophies.
    • Place of Presentation
      NC, U.S.A.
    • Year and Date
      2015-04-16
    • Related Report
      2015 Annual Research Report
    • Int'l Joint Research / Invited
  • [Presentation] 神経・筋疾患の分子メカニズム、遺伝子治療、分子標的治療 / Molecular pathogenesis, genetic counseling, and molecular targeting therapy for neurological and muscular diseases.2014

    • Author(s)
      戸田達史
    • Organizer
      日本人類遺伝学会第59回大会
    • Place of Presentation
      東京
    • Year and Date
      2014-11-19 – 2014-11-22
    • Related Report
      2014 Annual Research Report
    • Invited
  • [Presentation] 福山型筋ジストロフィー症の遺伝子治療研究2014

    • Author(s)
      戸田達史
    • Organizer
      第32回全国筋ジストロフィー大阪大会
    • Place of Presentation
      大阪
    • Year and Date
      2014-10-17 – 2014-10-18
    • Related Report
      2014 Annual Research Report
    • Invited
  • [Presentation] A de novo mutation of the MYH7 gene in a large Chinese family with autosomal dominant myopathy.2014

    • Author(s)
      Tatsushi Toda, Hui Xiong, Tetsuya Oda, Kazuhiro Kobayashi, Shuo Wang, Wataru Satake, Hui Jiao, Yanling Yang, Yutaka Suzuki, Sumio Sugano, Xiru Wu.
    • Organizer
      19th international congress of the world muscle society
    • Place of Presentation
      Berlin, Germany
    • Year and Date
      2014-10-07 – 2014-10-11
    • Related Report
      2014 Annual Research Report
  • [Presentation] Impaired viability of muscle precursor cells in muscular dystrophy with glycosylation defects and amelioration of its severe phenotype by limited gene expression.2014

    • Author(s)
      Motoi Kanagawa, Chih-Chieh Yu, Chiyomi Ito, So-ichiro Fukada, Tomoko Chiyo, Kazuhiro Kobayashi, Takashi Okada, Shin’ichi Takeda, Tatsushi Toda.
    • Organizer
      The 13th International Congress on Neuromuscular Diseases (ICNMD)
    • Place of Presentation
      Nice, France
    • Year and Date
      2014-07-05 – 2014-07-10
    • Related Report
      2014 Annual Research Report
  • [Presentation] 神経難病の治療と研究の現状と展望2014

    • Author(s)
      戸田 達史
    • Organizer
      第38回日本遺伝カウンセリング学会学術集会
    • Place of Presentation
      近畿大学ノーベンバーホール
    • Year and Date
      2014-06-26 – 2014-06-29
    • Related Report
      2014 Annual Research Report
    • Invited
  • [Presentation] 福山型筋ジストロフィーの病態基盤と新たな治療法の開発2014

    • Author(s)
      戸田 達史
    • Organizer
      第56回日本小児神経学会学術大会
    • Place of Presentation
      浜松
    • Year and Date
      2014-05-28 – 2014-05-31
    • Related Report
      2014 Annual Research Report
    • Invited
  • [Remarks] 神戸大学大学院 医学研究科 生理学・細胞生物学講座 分子脳科学分野

    • URL

      http://www.med.kobe-u.ac.jp/clgene/

    • Related Report
      2014 Annual Research Report
  • [Patent(Industrial Property Rights)] 筋ジストロフィー関連心筋症の治療又は予防剤2018

    • Inventor(s)
      片野坂 友紀、成瀬 恵治、氏原 嘉洋、戸田 達史、金川 基
    • Industrial Property Rights Holder
      片野坂 友紀、成瀬 恵治、氏原 嘉洋、戸田 達史、金川 基
    • Industrial Property Rights Type
      特許
    • Filing Date
      2018
    • Related Report
      2016 Annual Research Report
  • [Patent(Industrial Property Rights)] ジストログリカン糖鎖修飾異常に伴う疾患の治療剤及び関連酵素測定法2016

    • Inventor(s)
      戸田達史、小林千浩、金川基、遠藤玉夫、萬谷博、和田芳直、田尻道子
    • Industrial Property Rights Holder
      戸田達史、小林千浩、金川基、遠藤玉夫、萬谷博、和田芳直、田尻道子
    • Industrial Property Rights Type
      特許
    • Filing Date
      2016
    • Related Report
      2016 Annual Research Report
  • [Patent(Industrial Property Rights)] ジストログリカン糖鎖修飾異常に伴う疾患の治療剤2016

    • Inventor(s)
      戸田 達史、小林 千浩、金川 基、他
    • Industrial Property Rights Holder
      国立大学法人神戸大学、他
    • Industrial Property Rights Type
      特許
    • Industrial Property Number
      2016-160390
    • Filing Date
      2016-08-18
    • Related Report
      2015 Annual Research Report

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Published: 2014-04-04   Modified: 2019-03-29  

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