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Development of new gene therapies for epidermolysis bullosa

Research Project

Project/Area Number 22591215
Research Category

Grant-in-Aid for Scientific Research (C)

Allocation TypeSingle-year Grants
Section一般
Research Field Dermatology
Research InstitutionHokkaido University

Principal Investigator

OTA Mitsuhito  北海道大学, 大学院・医学研究科, 非常勤講 師 (10514242)

Co-Investigator(Kenkyū-buntansha) ITO Kei  北海道大学, 大学院・医学研究科, 非常勤講師 (20421977)
ARITA Ken  北海道大学, 大学院・医学研究科, 非常勤講 師 (50374434)
Project Period (FY) 2010 – 2012
Project Status Completed (Fiscal Year 2012)
Budget Amount *help
¥4,550,000 (Direct Cost: ¥3,500,000、Indirect Cost: ¥1,050,000)
Fiscal Year 2012: ¥910,000 (Direct Cost: ¥700,000、Indirect Cost: ¥210,000)
Fiscal Year 2011: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000)
Fiscal Year 2010: ¥2,080,000 (Direct Cost: ¥1,600,000、Indirect Cost: ¥480,000)
Keywords皮膚科学 / 表皮水疱症 / 遺伝子治療 / 水疱 / レンチウイルス
Research Abstract

This study was aimed at developing a new therapy for epidermolysis bullosa, which is one of the severest congenital skin diseases. The study design was utilizing genetic technologies to introduce genes into blisters of disease model animals. Gene introduction was confirmed when the method was applied to cultured cells. However, when it comes to in vivo experiments, introduced genes were not detected in the injected sites. We modified the strategy in which cultured cells were injected into blisters of the disease model animals. We found that the injected cells were functional at the injected sites for at least several days after the treatment.

Report

(4 results)
  • 2012 Annual Research Report   Final Research Report ( PDF )
  • 2011 Annual Research Report
  • 2010 Annual Research Report
  • Research Products

    (44 results)

All 2014 2013 2012 2011 2010

All Journal Article (29 results) (of which Peer Reviewed: 29 results) Presentation (14 results) (of which Invited: 2 results) Book (1 results)

  • [Journal Article] Sweet's syndrome presenting with vegetative nodules on the hands: relationship to neutrophilic dermatosis of the dorsal hands.2014

    • Author(s)
      Mizuno O, Yanagi T, Baba K, Yamane N, Inokuma D, Ito K, Akiyama M, Shimizu H
    • Journal Title

      Int J Dermatol

      Volume: (in press) Issue: 1 Pages: e46-e48

    • DOI

      10.1111/j.1365-4632.2011.05352.x

    • Related Report
      2012 Final Research Report
    • Peer Reviewed
  • [Journal Article] An Erythematous Plaque on the Breast2013

    • Author(s)
      Koguchi H, Arita K, Nakazato S, Moriuchi R, Yamane N, Shinkuma S, Matsumura K, Shimizu H
    • Journal Title

      A Quiz.Acta Derm Venereol

      Volume: (in press) Issue: 6 Pages: 763-765

    • DOI

      10.2340/00015555-1527

    • Related Report
      2012 Final Research Report
    • Peer Reviewed
  • [Journal Article] Indomethacin for refractory infantile eosinophilic pustular folliculitis2013

    • Author(s)
      Toyonaga E, Abe R, Moriuchi R, Ito K, Abe Y, Shimizu H
    • Journal Title

      JAMA Dermatol

      Volume: 149 Issue: 3 Pages: 367-8

    • DOI

      10.1001/2013.jamadermatol.591

    • Related Report
      2012 Final Research Report
    • Peer Reviewed
  • [Journal Article] Sweet's syndrome presenting with vegetative nodules on the hands: relationship to neutrophilic dermatosis of the dorsal hands.2013

    • Author(s)
      Mizuno O
    • Journal Title

      Int J Dermatol

      Volume: 印刷中

    • Related Report
      2012 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Indomethacin for refractory infantile eosinophilic pustular folliculitis.2013

    • Author(s)
      Toyonaga E
    • Journal Title

      JAMA Dermatol

      Volume: 149 Pages: 367-8

    • Related Report
      2012 Annual Research Report
    • Peer Reviewed
  • [Journal Article] An Erythematous Plaque on the Breast: A Quiz.2013

    • Author(s)
      Koguchi H
    • Journal Title

      Acta Derm Venereol

      Volume: 印刷中

    • Related Report
      2012 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Type VII collagen deficiency causesdefective tooth enamel formation due to poor differentiation of ameloblasts2012

    • Author(s)
      Umemoto H, Akiyama M, Domon T, Nomura T, Shinkuma S, Ito K, Asaka T, Sawamura D, Uitto J, Uo M, Kitagawa Y, Shimizu H
    • Journal Title

      Am J Pathol

      Volume: 181 Issue: 5 Pages: 1659-71

    • DOI

      10.1016/j.ajpath.2012.07.018

    • Related Report
      2012 Final Research Report
    • Peer Reviewed
  • [Journal Article] Erythema Annulare centrifugumu-like Neutrophilic Dermatosis : Effects of Potassium lodide2012

    • Author(s)
      Koguchi H, Arita K, et al
    • Journal Title

      Acta Derm Venereol

      Volume: 26 Issue: 3 Pages: 333-4

    • DOI

      10.2340/00015555-1291

    • Related Report
      2012 Final Research Report
    • Peer Reviewed
  • [Journal Article] Intraepidermal neutrophilic IgA pemphigus successfully treated with dapsone2012

    • Author(s)
      Hirata Y, Abe R, Kikuchi K, Hamasaka A, Shinkuma S, Nomura T, Nishie W, Arita K, Shimizu H
    • Journal Title

      Eur J Dermatol

      Volume: (in press) Issue: 2 Pages: 282-3

    • DOI

      10.1684/ejd.2012.1662

    • Related Report
      2012 Final Research Report
    • Peer Reviewed
  • [Journal Article] Skin involvement in ALK-negative systemic anaplastic large-cell lymphoma2012

    • Author(s)
      Hoshina D, Arita K, Mizuno O, Fujimoto K, Nishio M, Kondo T, Abe R, Shimizu H
    • Journal Title

      J Am Acad Dermatol

      Volume: 67 Issue: 4 Pages: e159-e160

    • DOI

      10.1016/j.jaad.2011.12.033

    • Related Report
      2012 Final Research Report
    • Peer Reviewed
  • [Journal Article] Type VII collagen deficiency causes defective tooth enamel formation due to poor differentiation of ameloblasts.2012

    • Author(s)
      Umemoto H
    • Journal Title

      Am J Pathol

      Volume: 181 Pages: 1659-71

    • Related Report
      2012 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Skin involvement in ALK-negative systemic anaplastic large-cell lymphoma.2012

    • Author(s)
      Hoshina D
    • Journal Title

      J Am Acad Dermatol

      Volume: 67

    • Related Report
      2012 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Intraepidermal neutrophilic IgA pemphigus successfully treated with dapsone.2012

    • Author(s)
      Hirata Y
    • Journal Title

      Eur J Dermatol

      Volume: 22 Pages: 282-3

    • Related Report
      2012 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Erythema annulare centrifugum-like neutrophilic dermatosis: effects of potassium iodide.2012

    • Author(s)
      Koguchi H
    • Journal Title

      Acta Derm Venereol

      Volume: 92 Pages: 333-4

    • Related Report
      2012 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Lichen planus in childhood showing various cutaneous features.2012

    • Author(s)
      Hamade Y
    • Journal Title

      Acta Derm Venereol

      Volume: 92 Pages: 386-7

    • Related Report
      2012 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Lichen Planus in childfood showing various cutaneous features2011

    • Author(s)
      Hamade Y, Arita K, et al
    • Journal Title

      Acta Derm Venereol

      Volume: 21 Issue: 4 Pages: 386-7

    • DOI

      10.2340/00015555-1260

    • Related Report
      2012 Final Research Report
    • Peer Reviewed
  • [Journal Article] Disseminated erythema multiforme-like reaction triggered by tinea unguium2011

    • Author(s)
      Tanimura S, Ota M
    • Journal Title

      Mycoses

      Volume: 54 Issue: 5

    • DOI

      10.1111/j.1439-0507.2010.01962.x

    • Related Report
      2012 Final Research Report 2011 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Childhood subepidermal blistering disease with autoantibodies against type VII collagen and laminin-3322011

    • Author(s)
      Lin HY, Yanagi T, Akiyama M, Iitani MM, Moriuchi R, Natsuga K, Shinkuma S, Yamane N, Inokuma D, Arita K, Shimizu H
    • Journal Title

      Br J Dermatol

      Volume: 164 Issue: 2 Pages: 452-454

    • DOI

      10.1111/j.1365-2133.2010.10065.x

    • Related Report
      2012 Final Research Report
    • Peer Reviewed
  • [Journal Article] A founder effect of c.1938delC in ITGB4 underlies junctional epidermolysis bullosa and its application for prenatal testing2011

    • Author(s)
      Natsuga K, Nishie W, Shinkuma S, Nakamura H, Ai-ita K, Yoneda K, Kusaka T, et al
    • Journal Title

      Exp Dermatol

      Volume: 20 Issue: 1 Pages: 74-6

    • DOI

      10.1111/j.1600-0625.2010.01177.x

    • Related Report
      2012 Final Research Report
    • Peer Reviewed
  • [Journal Article] Recurrence of hydroxyurea-induced leg ulcer after discontinuation of treatment/therapy2011

    • Author(s)
      Kikuchi K, Arita K, Tateishi Y, OnozawaM, Akiyama M, Shimizu H
    • Journal Title

      Acta Derm Venereol

      Volume: 91 Issue: 3 Pages: 373-374

    • DOI

      10.2340/00015555-1048

    • Related Report
      2012 Final Research Report
    • Peer Reviewed
  • [Journal Article] Disseminated erythema multiforme-like reaction triggered by tinea unauium.2011

    • Author(s)
      Tanimura S, Ota M
    • Journal Title

      Mycoses

      Volume: (In press)

    • Related Report
      2010 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Self-healing congenital generalized skin creases: Michelin tire baby syndrome2010

    • Author(s)
      Nomura Y, Ota M, Tochimaru H
    • Journal Title

      J Am Acad Dermatol

      Volume: 63 Issue: 6 Pages: 1110-1111

    • DOI

      10.1016/j.jaad.2009.03.025

    • Related Report
      2012 Final Research Report
    • Peer Reviewed
  • [Journal Article] Ice cream urticaria2010

    • Author(s)
      Ota M, Kawasaki H, Horimoto M
    • Journal Title

      Am J Med

      Volume: 123 Issue: 12 Pages: e1-e2

    • DOI

      10.1016/j.amjmed.2010.04.034

    • Related Report
      2012 Final Research Report
    • Peer Reviewed
  • [Journal Article] Prevalent LIPH founder mutations lead to loss of P2Y5 activation ability of PA-PLA1alpha in autosomal recessive hypotrichosis2010

    • Author(s)
      Shinkuma S, Akiyama M, Inoue A, Aoki J, Natsuga K, Nomura T, Arita K, Abe R, Ito K, Nakamura H, Ujiie H, Shibaki A, Suga H, Tsunemi Y, Nishie W, Shimizu H
    • Journal Title

      Hum Mutat

      Volume: 31 Pages: 602-10

    • DOI

      10.1002/humu.21235

    • Related Report
      2012 Final Research Report
    • Peer Reviewed
  • [Journal Article] Novel IL31RA gene mutation and ancestral OSMR mutant allele in familial primary cutaneous amyloidosis.2010

    • Author(s)
      Lin MW, Lee DD, Liu TT, Lin YF, Chen SY, Huang CC, Weng HY, Liu YF, Tanaka A, Arita K, Lai-Cheong J, Palisson F, Chang YT, Wong CK, Matsuura I, McGrath JA, Tsai SF
    • Journal Title

      Eur J Hum Genet

      Volume: 18 Issue: 1 Pages: 26-32

    • DOI

      10.1038/ejhg.2009.135

    • Related Report
      2012 Final Research Report
    • Peer Reviewed
  • [Journal Article] Complete paternal isodisomy of chromosome17 in junctional epidermolysis bullosa with pyloric atresia2010

    • Author(s)
      Natsuga K, Nishie W, Arita K, Shinkuma S, Nakamura H, Kubota S, Imakado S, Akiyama M, Shimizu H
    • Journal Title

      J Invest Dermatol

      Volume: 130 Issue: 11 Pages: 2671-2674

    • DOI

      10.1038/jid.2010.182

    • Related Report
      2012 Final Research Report
    • Peer Reviewed
  • [Journal Article] Plectin deficiency leads to both muscular dystrophy and pyloric atresia in epidermolysis bullosa simplex2010

    • Author(s)
      Natsuga K, Nishie W, Shinkuma S, Arita K, Nakamura H, Ohyama M, Osaka H, Kambara T, Hirako Y, Shimizu H
    • Journal Title

      Hum Mutat

      Volume: 31 Issue: 10 Pages: E1687-E1698

    • DOI

      10.1002/humu.21330

    • Related Report
      2012 Final Research Report
    • Peer Reviewed
  • [Journal Article] Ice cream urticaria.2010

    • Author(s)
      Ota M, Kawasaki H, et al.
    • Journal Title

      Am J Med

      Volume: 123

    • Related Report
      2010 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Self-healing congenital generalized skin creases : Michelin tire baby syndrome.2010

    • Author(s)
      Nomura Y, Ota M, et al.
    • Journal Title

      J Am Acad Dermatol

      Volume: 63 Pages: 1110-1111

    • Related Report
      2010 Annual Research Report
    • Peer Reviewed
  • [Presentation] アトピー性皮膚炎~最近の話題と寛解維持に導くための標準治療2012

    • Author(s)
      大田光仁
    • Organizer
      千歳恵庭小児科学術講演会
    • Place of Presentation
      千歳、千歳ANAクラウンホテル
    • Year and Date
      2012-07-18
    • Related Report
      2012 Final Research Report
  • [Presentation] 皮膚からはじまる食物アレルギー2012

    • Author(s)
      大田光仁
    • Organizer
      千歳市民健康講座
    • Place of Presentation
      千歳市立千歳市民病院
    • Year and Date
      2012-05-12
    • Related Report
      2012 Final Research Report
  • [Presentation] 乾癬に対する生物学的製剤治療の光と影~当科80例の経験を通じて2012

    • Author(s)
      伊藤圭,飯谷麻里,大宮若菜,渡邉美佳
    • Organizer
      第42回日本創傷治癒学会.
    • Place of Presentation
      札幌
    • Related Report
      2012 Final Research Report
  • [Presentation] どの生物学的製剤を選択すべきか~「正解なき薬剤選択」を含めた乾癬治療戦略2012

    • Author(s)
      伊藤圭
    • Organizer
      第76回日本皮膚科学会東部支部学術大会
    • Place of Presentation
      札幌,ロイトン札幌
    • Related Report
      2012 Final Research Report
  • [Presentation] ウステキヌマブの広く高い有用性~認可1年半の当科使用経験を通じて2012

    • Author(s)
      伊藤圭
    • Organizer
      第27回日本乾癬学会学術大会.
    • Place of Presentation
      新潟、朱鷺メッセ
    • Related Report
      2012 Final Research Report
  • [Presentation] ウステキヌマブの広く 高い有用性 ~認可1年半の当科使用経験を通じて~2012

    • Author(s)
      伊藤圭
    • Organizer
      第27回日本乾癬学会学術大会
    • Place of Presentation
      朱鷺メッセ(新潟)
    • Related Report
      2012 Annual Research Report
    • Invited
  • [Presentation] どの生物学的製剤を選 択すべきか ~「正解なき薬剤選択」を含めた乾癬治 療戦略~.2012

    • Author(s)
      伊藤圭
    • Organizer
      第76回日本皮膚科学会東部支部学術大会
    • Place of Presentation
      ロイトン札幌(北海道)
    • Related Report
      2012 Annual Research Report
    • Invited
  • [Presentation] 乾癬に対する生物学的製剤治療の光と影 ~当科80 例の経験を通じて~2012

    • Author(s)
      伊藤圭
    • Organizer
      第42回日本創傷治癒学会
    • Place of Presentation
      かでる2.7(北海道)
    • Related Report
      2012 Annual Research Report
  • [Presentation] インフリキシマブ投与乾癬患者25例およびバイオロジクススイッチ5症例を含むウステキヌマブ投与患者20例の使用経験2011

    • Author(s)
      伊藤圭,飯谷麻里,渡邊英里香,大宮若菜,豊永愛恋,小玉和郎,小林仁,安田秀美,浜坂幸吉,村本文男,大田光仁,西村真智子
    • Organizer
      第388回日本皮膚科学会北海道地方会
    • Place of Presentation
      札幌、札幌医科大学講堂
    • Year and Date
      2011-12-17
    • Related Report
      2012 Final Research Report
  • [Presentation] 乾癬への生物学的製剤認可1年半の当院での治療の軌跡2011

    • Author(s)
      伊藤圭,飯谷麻里,渡邊英里香,大宮若菜
    • Organizer
      第91回日本交通医学会北海道地方会札幌
    • Place of Presentation
      JR札幌病院講堂
    • Year and Date
      2011-09-03
    • Related Report
      2012 Final Research Report
  • [Presentation] 7q22.1 as a possible gene locus of Michelin tire baby syndrome2011

    • Author(s)
      Arita K, Nomura T, Nomura Y, Ota M, Shimizu H
    • Organizer
      The 36th annual meeting of the Japanese Society for Investigative Dermatology Kyoto
    • Place of Presentation
      京都国際会館
    • Related Report
      2012 Final Research Report
  • [Presentation] Cosmetic lip tattoo により生じた広範な乾酪壊死巣を伴う口唇全周性 granulomatous reaction の 1 例2011

    • Author(s)
      伊藤圭,飯谷麻里,渡邊英里香,大宮若菜,小林仁,佐々木真由美
    • Organizer
      第63回日本皮膚科学会西部支部学術大会
    • Place of Presentation
      沖縄コンベンションセンター
    • Related Report
      2012 Final Research Report
  • [Presentation] インフリキシマブ投与乾癬患者22例および切り替え5症例を含むウステキヌマブ投与患者10例の使用経験2011

    • Author(s)
      伊藤圭,飯谷麻里,渡邊英里香,大宮若菜,豊永愛恋,小玉和郎,小林仁,安田秀美,浜坂幸吉,村本文男,西村真智子
    • Organizer
      第26回日本乾癬学会学術大会大阪
    • Place of Presentation
      大阪国際会議場
    • Related Report
      2012 Final Research Report
  • [Presentation] 日本人に生じたDuhring疱疹状皮膚炎における表皮トランスグルタミーゼ(eTG)抗体価の検討2010

    • Author(s)
      新熊悟, 大田光仁, 他
    • Organizer
      第384回日本皮膚科学会北海道地方会
    • Place of Presentation
      札幌医大、札幌市
    • Year and Date
      2010-12-11
    • Related Report
      2010 Annual Research Report
  • [Book] 脛骨前粘液水腫(甲状腺機能亢進症)2011

    • Author(s)
      伊藤圭
    • Total Pages
      93
    • Publisher
      メディカルレビュー社
    • Related Report
      2012 Final Research Report

URL: 

Published: 2010-08-23   Modified: 2019-07-29  

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