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Development of a novel gene therapy method via CRISPR/Cas9 mediated in vivo genome editing

Research Project

Project/Area Number 15K18330
Research Category

Grant-in-Aid for Young Scientists (B)

Allocation TypeMulti-year Fund
Research Field Neurophysiology / General neuroscience
Research InstitutionGunma University

Principal Investigator

Konno Ayumu  群馬大学, 大学院医学系研究科, 講師 (40509048)

Project Period (FY) 2015-04-01 – 2018-03-31
Project Status Completed (Fiscal Year 2017)
Budget Amount *help
¥4,290,000 (Direct Cost: ¥3,300,000、Indirect Cost: ¥990,000)
Fiscal Year 2017: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
Fiscal Year 2016: ¥1,300,000 (Direct Cost: ¥1,000,000、Indirect Cost: ¥300,000)
Fiscal Year 2015: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000)
Keywords遺伝子治療 / アデノ随伴ウイルスベクター / CRISPR/Cas9 / ゲノム編集 / 脊髄小脳失調症 / AAV
Outline of Final Research Achievements

In this study, we aimed to develop a novel gene therapy method for spinocerebellar ataxia type 1 with CRISPR/Cas9 mediated in vivo genome editing using a gene delivery by adeno-associated virus vector. Although we examined SpCas9 and SaCas9 for in vivo knockout, we could not observe the amelioration of an ataxia in a mouse model of spinocerebellar ataxia type 1. Further investigations for higher efficiencies of a gene expression and/or a gene editing are needed.

Report

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

    (26 results)

All 2018 2017 2016 2015 Other

All Int'l Joint Research (2 results) Journal Article (14 results) (of which Int'l Joint Research: 7 results,  Peer Reviewed: 14 results,  Open Access: 9 results,  Acknowledgement Compliant: 3 results) Presentation (5 results) (of which Int'l Joint Research: 1 results) Remarks (5 results)

  • [Int'l Joint Research] University of Oxford(United Kingdom)

    • Related Report
      2017 Annual Research Report
  • [Int'l Joint Research] Trustees of Dartmouth College/University Tenessee/Max Planck Florida Institute(米国)

    • Related Report
      2017 Annual Research Report
  • [Journal Article] Intravenous administration of the adeno-associated virus-PHP.B capsid fails to upregulate transduction efficiency in the marmoset brain2018

    • Author(s)
      Matsuzaki Yasunori、Konno Ayumu、Mochizuki Ryuta、Shinohara Yoichiro、Nitta Keisuke、Okada Yukihiro、Hirai Hirokazu
    • Journal Title

      Neuroscience Letters

      Volume: 665 Pages: 182-188

    • DOI

      10.1016/j.neulet.2017.11.049

    • Related Report
      2017 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Elavl3 is essential for the maintenance of Purkinje neuron axons2018

    • Author(s)
      Ogawa Yuki、Kakumoto Kyoko、Yoshida Tetsu、Kuwako Ken-ichiro、Miyazaki Taisuke、Yamaguchi Junji、Konno Ayumu、Hata Junichi、Uchiyama Yasuo、Hirai Hirokazu、Watanabe Masahiko、Darnell Robert B.、Okano Hideyuki、Okano Hirotaka James
    • Journal Title

      Sci Rep.

      Volume: 8 Issue: 1 Pages: 2722-2722

    • DOI

      10.1038/s41598-018-21130-5

    • Related Report
      2017 Annual Research Report
    • Peer Reviewed / Open Access / Int'l Joint Research
  • [Journal Article] Viral Vector-Based Evaluation of Regulatory Regions in the Neuron-Specific Enolase (NSE) Promoter in Mouse Cerebellum In Vivo2017

    • Author(s)
      Shinohara Yoichiro、Ohtani Toshinori、Konno Ayumu、Hirai Hirokazu
    • Journal Title

      The Cerebellum

      Volume: 16 Issue: 5-6 Pages: 913-922

    • DOI

      10.1007/s12311-017-0866-5

    • Related Report
      2017 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Red fluorescent protein-based cAMP indicator applicable to optogenetics and in vivo imaging2017

    • Author(s)
      Harada Kazuki、Ito Motoki、Wang Xiaowen、Tanaka Mika、Wongso Devina、Konno Ayumu、Hirai Hirokazu、Hirase Hajime、Tsuboi Takashi、Kitaguchi Tetsuya
    • Journal Title

      Scientific Reports

      Volume: 7 Issue: 1 Pages: 7351-7351

    • DOI

      10.1038/s41598-017-07820-6

    • Related Report
      2017 Annual Research Report
    • Peer Reviewed / Open Access / Int'l Joint Research
  • [Journal Article] Minimal Purkinje Cell-Specific PCP2/L7 Promoter Virally Available for Rodents and Non-human Primates2017

    • Author(s)
      Nitta Keisuke、Matsuzaki Yasunori、Konno Ayumu、Hirai Hirokazu
    • Journal Title

      Molecular Therapy - Methods & Clinical Development

      Volume: 6 Pages: 159-170

    • DOI

      10.1016/j.omtm.2017.07.006

    • Related Report
      2017 Annual Research Report
    • Peer Reviewed / Open Access
  • [Journal Article] Regulatory connection between the expression level of classical protein kinase C and pruning of climbing fibers from cerebellar Purkinje cells2017

    • Author(s)
      Takahashi Nobutaka、Shuvaev Anton N.、Konno Ayumu、Matsuzaki Yasunori、Watanave Masashi、Hirai Hirokazu
    • Journal Title

      Journal of Neurochemistry

      Volume: 143 Issue: 6 Pages: 660-670

    • DOI

      10.1111/jnc.14239

    • Related Report
      2017 Annual Research Report
    • Peer Reviewed / Int'l Joint Research
  • [Journal Article] Inflammation-induced reversible switch of the neuron-specific enolase promoter from Purkinje neurons to Bergmann glia.2016

    • Author(s)
      Sawada Y, Konno A, Nagaoka J, Hirai H
    • Journal Title

      Scientific Reports

      Volume: 6 Issue: 1 Pages: 27758-27758

    • DOI

      10.1038/srep27758

    • Related Report
      2016 Research-status Report
    • Peer Reviewed / Open Access
  • [Journal Article] Fluorescent-based evaluation of chaperone-mediated autophagy and microautophagy activities in cultured cells.2016

    • Author(s)
      Sato M, Seki T, Konno A, Hirai H, Kurauchi Y, Hisatsune A, Katsuki H.
    • Journal Title

      Genes to Cells

      Volume: 21 Issue: 8 Pages: 861-873

    • DOI

      10.1111/gtc.12390

    • Related Report
      2016 Research-status Report
    • Peer Reviewed / Open Access / Acknowledgement Compliant
  • [Journal Article] Viral Vector-Based Dissection of Marmoset GFAP Promoter in Mouse and Marmoset Brains.2016

    • Author(s)
      Shinohara Y, Konno A, Takahashi N, Matsuzaki Y, Kishi S, Hirai H
    • Journal Title

      PLoS ONE

      Volume: 11 Issue: 8 Pages: e0162023-e0162023

    • DOI

      10.1371/journal.pone.0162023

    • Related Report
      2016 Research-status Report
    • Peer Reviewed / Open Access / Acknowledgement Compliant
  • [Journal Article] FMRP Expression Levels in Mouse CNS Neurons Determine Behavioral Phenotype.2016

    • Author(s)
      Arsenault J, Gholizadeh S, Niibori Y, Pacey LK, Halder SK, Koxhioni E, Konno A, Hirai H, Hampson DR
    • Journal Title

      Human Gene Therapy

      Volume: 27 Issue: 12 Pages: 982-996

    • DOI

      10.1089/hum.2016.090

    • Related Report
      2016 Research-status Report
    • Peer Reviewed / Open Access / Int'l Joint Research
  • [Journal Article] Fusion of Human Fetal Mesenchymal Stem Cells with “Degenerating” Cerebellar Neurons in Spinocerebellar Ataxia Type 1 Model Mice.2016

    • Author(s)
      Huda F, Fan Y, Suzuki M, Konno A, Matsuzaki Y, Takahashi N, Chan JK, Hirai H
    • Journal Title

      PLoS ONE

      Volume: 11 Issue: 11 Pages: e0164202-e0164202

    • DOI

      10.1371/journal.pone.0164202

    • Related Report
      2016 Research-status Report
    • Peer Reviewed / Open Access / Int'l Joint Research
  • [Journal Article] Transduction Profile of the Marmoset Central Nervous System Using Adeno-Associated Virus Serotype 9 Vectors2016

    • Author(s)
      Matsuzaki Y, Konno A, Mukai R, Honda F, Hirato M, Yoshimoto Y, Hirai H.
    • Journal Title

      Molecular Neurobiology

      Volume: in press Issue: 3 Pages: 1-16

    • DOI

      10.1007/s12035-016-9777-6

    • Related Report
      2015 Research-status Report
    • Peer Reviewed / Int'l Joint Research
  • [Journal Article] Calcium imaging reveals glial involvement in transcranial direct current stimulation-induced plasticity in mouse brain2016

    • Author(s)
      H Monai, M Ohkura, M Tanaka, Y Oe, A Konno, H Hirai, K Mikoshiba, S Itohara, J Nakai, Y Iwai & H Hirase
    • Journal Title

      Nature communications

      Volume: 7 Issue: 1 Pages: 11100-11100

    • DOI

      10.1038/ncomms11100

    • Related Report
      2015 Research-status Report
    • Peer Reviewed / Open Access / Int'l Joint Research / Acknowledgement Compliant
  • [Journal Article] Plasticity of the developmentally arrested staggerer cerebellum in response to exogenous RORα.2015

    • Author(s)
      Iizuka A, Matsuzaki Y, Konno A, Hirai H.
    • Journal Title

      Brain Structure and Function

      Volume: in press Issue: 6 Pages: 1-11

    • DOI

      10.1007/s00429-015-1077-9

    • Related Report
      2015 Research-status Report
    • Peer Reviewed
  • [Presentation] In vivo evaluation of the shortened-WPRE in the cerebellum2017

    • Author(s)
      Ayumu Konno, Kazuma Hachisu, Hirokazu Hirai
    • Organizer
      第40回日本神経科学大会
    • Related Report
      2017 Annual Research Report
  • [Presentation] アデノ随伴ウイルスによる遺伝子導入を介したリピート病モデル動物の作出と治療法の開発2017

    • Author(s)
      今野 歩, 渡邊 将, 星野 千秋, 篠原 洋一郎, 松﨑 泰教, 平井 宏和
    • Organizer
      2017年度生命科学系学会合同年次大会(ConBio2017)
    • Related Report
      2017 Annual Research Report
  • [Presentation] Generation of animal models of spinocerebellar ataxia by AAV9-mediated gene delivery2016

    • Author(s)
      Ayumu Konno, Yasunori Matsuzaki & Hirokazu Hirai
    • Organizer
      第93回日本生理学会大会
    • Place of Presentation
      札幌コンベンションセンター/北海道札幌市
    • Year and Date
      2016-03-22
    • Related Report
      2015 Research-status Report
  • [Presentation] AAV9 vector-mediated production of animal models of spinocerebellar ataxia2016

    • Author(s)
      Ayumu Konno, Yasunori Matsuzaki & Hirokazu Hirai
    • Organizer
      International Society of Radiation Neurobiology 6th Conference
    • Place of Presentation
      良順会館(長崎大学医学部坂本キャンパス内)/長崎県長崎市
    • Year and Date
      2016-02-12
    • Related Report
      2015 Research-status Report
    • Int'l Joint Research
  • [Presentation] A marmoset model of spinocerebellar ataxia type 3 generated using AAV vectors2015

    • Author(s)
      Ayumu Konno, Yasunori Matsuzaki, Yoichiro Shinohara, and Hirokazu Hirai
    • Organizer
      第38回日本神経科学大会
    • Place of Presentation
      神戸国際会議場/展示場・兵庫県神戸市
    • Year and Date
      2015-07-28
    • Related Report
      2015 Research-status Report
  • [Remarks] 群馬大学大学院医学系研究科脳神経再生医学Web site

    • URL

      http://synapse.dept.med.gunma-u.ac.jp/

    • Related Report
      2017 Annual Research Report
  • [Remarks] 赤色 cAMP 可視化蛍光タンパク質センサーの開発

    • URL

      http://www.c.u-tokyo.ac.jp/info/news/topics/files/20170810pressrelease.pdf

    • Related Report
      2017 Annual Research Report
  • [Remarks] 群馬大学大学院医学系研究科 脳神経再生医学分野

    • URL

      http://synapse.dept.med.gunma-u.ac.jp/

    • Related Report
      2016 Research-status Report
  • [Remarks] 群馬大学大学院医学系研究科脳神経再生医学分野

    • URL

      http://synapse.dept.med.gunma-u.ac.jp/

    • Related Report
      2015 Research-status Report
  • [Remarks] 微弱な電気刺激が脳を活性化する仕組みを解明

    • URL

      http://www.riken.jp/pr/press/2016/20160322_1/

    • Related Report
      2015 Research-status Report

URL: 

Published: 2015-04-16   Modified: 2022-06-07  

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