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Development of disease monkey model for hereditary retinal disease by gene editing

Research Project

Project/Area Number 15K15641
Research Category

Grant-in-Aid for Challenging Exploratory Research

Allocation TypeMulti-year Fund
Research Field Ophthalmology
Research Institution独立行政法人国立病院機構(東京医療センター臨床研究センター)

Principal Investigator

Iwata Takeshi  独立行政法人国立病院機構(東京医療センター臨床研究センター), 分子細胞生物学研究部, 部長 (90374157)

Co-Investigator(Kenkyū-buntansha) MIZOTA Atsushi  帝京大学, 医学部, 教授 (10239262)
SHIMOZAWA Nobuhiro  医薬基盤研究所, 霊長類医科学研究センター, 主任研究員 (50300786)
Project Period (FY) 2015-04-01 – 2016-03-31
Project Status Completed (Fiscal Year 2015)
Budget Amount *help
¥3,640,000 (Direct Cost: ¥2,800,000、Indirect Cost: ¥840,000)
Fiscal Year 2015: ¥3,640,000 (Direct Cost: ¥2,800,000、Indirect Cost: ¥840,000)
Keywords医歯薬学 / 外科系臨床医学 / 眼科学 / 眼生化学・分子生物学 / カニクイザル / 霊長類 / CRISPR/Cas9 / ノックアウト / 遺伝子改変 / 疾患モデル動物
Outline of Final Research Achievements

The primate eye has unique structural characteristic such as macula, which do not exist in experimental animals, mouse, rat, and zebrafish. In this study, CRISPR/Cas9 was used to specifically cut the genome DNA in the fertilized egg to develop gene manipulated cynomolgus macaque monkeys with hereditary retinal disease. The fertilized egg was injected with CRISPR/Cas9 vector to specifically cut gene responsible for inherited macula disease. ES cell were detected with DNA cleavage at the site targeted. The detection of targeted mutation in ES cells provides the possibility of new born mutation by continuation of further experiments. A single offspring was born recently born. The new methods of CRISPR/Cas9 will be used to create other inherited macula diseases.

Report

(2 results)
  • 2015 Annual Research Report   Final Research Report ( PDF )
  • Research Products

    (7 results)

All 2015

All Journal Article (7 results) (of which Peer Reviewed: 7 results,  Open Access: 1 results,  Acknowledgement Compliant: 1 results)

  • [Journal Article] HTRA1 Overexpression Induces the Exudative Form of Age-related Macular Degeneration.2015

    • Author(s)
      Iejima D, Nakayama M, Iwata T
    • Journal Title

      Nova Science Publishers

      Volume: 10 Pages: 193-203

    • Related Report
      2015 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Congenital Achromatopsia and Macular Atrophy Caused by a Novel Recessive PDE6C Mutation (p.E591K).2015

    • Author(s)
      Katagiri S, Hayashi T, Yoshitake K, Sergeev Y, Akahori M, Furuno M, Nishino J, Ikeo K, Tsunoda K, Tsuneoka H, Iwata T.
    • Journal Title

      Ophthalmic Genet.

      Volume: Jun;36(2) Issue: 2 Pages: 137-44

    • DOI

      10.3109/13816810.2014.991932

    • Related Report
      2015 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Association of Retinal Artery and Other Inner Retinal Structures With Distribution of Tapetal-like Reflex in Oguchi's Disease.2015

    • Author(s)
      Kato Y, Tsunoda K, Fujinami K, Iwata T, Saga M, Oguchi Y.
    • Journal Title

      Invest Ophthalmol Vis Sci.

      Volume: Apr;56(4) Issue: 4 Pages: 2162-72

    • DOI

      10.1167/iovs.14-16198

    • Related Report
      2015 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Author Response: Postnatal Overexpression of the Human ARMS2 Gene Does Not Induce Abnormalities in Retina and Choroid in Transgenic Mouse Models.2015

    • Author(s)
      Iwata T.
    • Journal Title

      Invest Ophthalmol Vis Sci.

      Volume: 56 Issue: 2 Pages: 1389-1389

    • DOI

      10.1167/iovs.14-16174

    • Related Report
      2015 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Reduced rod electroretinograms in carrier parents of two Japanese siblings with autosomal recessive retinitis pigmentosa associated with PDE6B gene mutations.2015

    • Author(s)
      Kuniyoshi K, Sakuramoto H, Yoshitake K, Ikeo K, Furuno M, Tsunoda K, Kusaka S, Shimomura Y, Iwata T.
    • Journal Title

      Doc Ophthalmol.

      Volume: Aug;131(1) Issue: 1 Pages: 71-9

    • DOI

      10.1007/s10633-015-9497-7

    • Related Report
      2015 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Pathologic Changes of Cone Photoreceptors in Eyes With Occult Macular Dystrophy.2015

    • Author(s)
      Nakanishi A, Ueno S, Kawano K, Ito Y, Kominami T, Yasuda S, Kondo M, Tsunoda K, Iwata T, Terasaki H.
    • Journal Title

      Invest Ophthalmol Vis Sci.

      Volume: Nov;56(12) Issue: 12 Pages: 7243-9

    • DOI

      10.1167/iovs.15-16742

    • Related Report
      2015 Annual Research Report
    • Peer Reviewed / Open Access
  • [Journal Article] Mutation analysis of BEST1 in Japanese patients with Best’s vitelliform macular dystrophy2015

    • Author(s)
      Katagiri S,Hayashi T,Ohkuma Y,Sekiryu T,Takeuchi T,Gekka T,et al.
    • Journal Title

      Br J Ophthalmol

      Volume: 99 Issue: 11 Pages: 1577-1582

    • DOI

      10.1136/bjophthalmol-2015-306830

    • Related Report
      2015 Annual Research Report
    • Peer Reviewed / Acknowledgement Compliant

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Published: 2015-04-16   Modified: 2017-05-10  

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