UCHIYAMA Toru 内山徹

Researcher Number	10436107
Affiliation (Current)	2025: 国立研究開発法人国立成育医療研究センター, 成育遺伝研究部, 室長
Affiliation (based on the past Project Information) *help	2015 – 2024: 国立研究開発法人国立成育医療研究センター, 成育遺伝研究部, 室長 2014: 独立行政法人国立成育医療研究センター, その他部局等, その他 2014: 独立行政法人国立成育医療研究センター, 成育遺伝研究部, 室長 2013: 独立行政法人国立成育医療研究センター, その他部局等, 室長 2012: 東北大学, 病院, 助教 2011 – 2012: 東北大学, 大学病院, 助教
Review Section/Research Field	Principal Investigator Pediatrics / Basic Section 52050:Embryonic medicine and pediatrics-related Except Principal Investigator Medium-sized Section 52:General internal medicine and related fields / Pediatrics / Basic Section 56040:Obstetrics and gynecology-related / Basic Section 52050:Embryonic medicine and pediatrics-related / Tumor therapeutics
Keywords	Principal Investigator 遺伝子治療 / ゲノム編集 / 原発性免疫不全症 / STAT3 / 高IgE症候群 / 変異アリル特異的 / Cas9 / 変異アレル特異的編集 / 機能獲得型変異 / ウイルス安定産生細胞株 … More / 安定ウイルス産生細胞株 / baboon envelope / ヒト造血幹細胞 / BaEV / ヒヒ内在性レトロウイルス / 幹細胞性 / 造血幹細胞遺伝子治療 / レンチウイルスベクター / dNLnk / X連鎖重症複合免役不全症 / X連鎖重症複合免疫不全症 / FVベクター / ADA欠損症 / 相同組み替え / 造血幹細胞 / iPS細胞 / 相同組換え … More Except Principal Investigator 癌 / 遺伝子治療 / ドラックデリバリー / c-kit / レンチウイルスベクター / 薬物送達 / 造血幹細胞 / エンハンサー / スーパーエンハンサー / エンハンサー・プロモーター相互作用 / 子宮内膜 / 胎盤 / クロマチン高次構造 / 白血病 / 再生医学 / 免疫学 / 遺伝子 / 固形癌 / 癌免疫療法 / 遺伝子細胞療法 / 癌精巣抗原 / CAR-T 細胞療法 / OTC欠損症 / iPS細胞 / OTC 欠損症 / iPS 細胞 / 遺伝子修復 Less

特異的リガンドを使用した造血幹細胞へのin vivo遺伝子送達ベクター開発
- Principal Investigator
  
  内田直也
- Project Period (FY)
  2023 – 2025
- Research Category
  
  Grant-in-Aid for Challenging Research (Pioneering)
- Review Section
  
  Medium-sized Section 52:General internal medicine and related fields
- Research Institution
  The University of Tokyo
Development of a novel gene therapy strategy using base editing for hyper-IgE syndrome caused by STAT3 mutationsPrincipal Investigator
- Principal Investigator
  
  内山徹
- Project Period (FY)
  2022 – 2024
- Research Category
  
  Grant-in-Aid for Scientific Research (C)
- Review Section
  
  Basic Section 52050:Embryonic medicine and pediatrics-related
- Research Institution
  National Center for Child Health and Development
Elucidation of 3D genome architecture of human placental and endometrial cells
- Principal Investigator
  
  中林一彦
- Project Period (FY)
  2022 – 2024
- Research Category
  
  Grant-in-Aid for Scientific Research (B)
- Review Section
  
  Basic Section 56040:Obstetrics and gynecology-related
- Research Institution
  National Center for Child Health and Development
Development of a hematopoietic stem cell-targeted lentiviral vector system
- Principal Investigator
  
  内田直也
- Project Period (FY)
  2021 – 2022
- Research Category
  
  Fund for the Promotion of Joint International Research (Fostering Joint International Research (B))
- Review Section
  
  Medium-sized Section 52:General internal medicine and related fields
- Research Institution
  The University of Tokyo
Investigation of molecular biology of CNS leukemia
- Principal Investigator
  
  Kato Motohiro
- Project Period (FY)
  2020 – 2022
- Research Category
  
  Grant-in-Aid for Scientific Research (B)
- Review Section
  
  Basic Section 52050:Embryonic medicine and pediatrics-related
- Research Institution
  The University of Tokyo
Genome editing-based gene therapy for primary immunodeficiency caused by dominant gain-of-function mutations.Principal Investigator
- Principal Investigator
  
  Uchiyama Toru
- Project Period (FY)
  2019 – 2021
- Research Category
  
  Grant-in-Aid for Scientific Research (C)
- Review Section
  
  Basic Section 52050:Embryonic medicine and pediatrics-related
- Research Institution
  National Center for Child Health and Development
Development of novel CAR-T cell therapy using a cancer-testis antigen CRT2
- Principal Investigator
  
  Hayashi Emiko
- Project Period (FY)
  2017 – 2020
- Research Category
  
  Grant-in-Aid for Scientific Research (C)
- Research Field
  
  Tumor therapeutics
- Research Institution
  Nippon Medical School
  National Center for Child Health and Development
Comprehensive analysis of somatic and germline genomic alterations for atypical leukemia in children
- Principal Investigator
  
  Kato Motohiro
- Project Period (FY)
  2017 – 2019
- Research Category
  
  Grant-in-Aid for Scientific Research (B)
- Research Field
  
  Pediatrics
- Research Institution
  National Center for Child Health and Development
Establishment of Baboon envelope pseudotyped LV vector system to maintain the stemness in stem cell gene therapy.Principal Investigator
- Principal Investigator
  
  Uchiyama Toru
- Project Period (FY)
  2015 – 2017
- Research Category
  
  Grant-in-Aid for Scientific Research (C)
- Research Field
  
  Pediatrics
- Research Institution
  National Center for Child Health and Development
Foamy virus vector-mediated gene transfer into long-term HSC in the gene therapy for primary immunodeficiencies.Principal Investigator
- Principal Investigator
  
  UCHIYAMA TORU
- Project Period (FY)
  2013 – 2014
- Research Category
  
  Grant-in-Aid for Young Scientists (B)
- Research Field
  
  Pediatrics
- Research Institution
  National Research Institute for Child Health and Development
Homologous recombination-mediated gene correction in iPS cells o Ornithine transcarbamylase deficiency
- Principal Investigator
  
  TSUCHIYA Shigeru
- Project Period (FY)
  2011 – 2012
- Research Category
  
  Grant-in-Aid for Challenging Exploratory Research
- Research Field
  
  Pediatrics
- Research Institution
  Tohoku University
Homologous recombination-mediated gene correction for Adenosine deaminase deficiencyPrincipal Investigator
- Principal Investigator
  
  UCHIYAMA Toru
- Project Period (FY)
  2011 – 2012
- Research Category
  
  Grant-in-Aid for Young Scientists (B)
- Research Field
  
  Pediatrics
- Research Institution
  Tohoku University

[Book] 先端治療技術の実用化と開発戦略2017
- Author(s)
  内山徹
- Total Pages
  7
- Publisher
  技術情報協会
- ISBN
  9784861046506
- Data Source
  KAKENHI-PROJECT-15K09676
[Book] 小児内科2017
- Author(s)
  内山徹
- Total Pages
  4
- Publisher
  東京医学社
- Data Source
  KAKENHI-PROJECT-15K09676
[Journal Article] Melting temperature mapping method in children: Rapid identification of pathogenic microbes.2024
- Author(s)
  Ishikawa T, Uejima Y, Okai M, Shiga K, Shoji K, Miyairi I, Kato M, Morooka S, Kubota M, Tagaya T, Tsuji S, Aoki S, Ide K, Niimi H, Uchiyama T, Onodera M, Kawai T.
- Journal Title
  
  J Infect Chemother.
  
  Volume: 30(6) Issue: 6 Pages: 475-480
- DOI
  10.1016/j.jiac.2023.11.024
- Peer Reviewed / Open Access
- Data Source
  KAKENHI-PROJECT-22K07951, KAKENHI-PROJECT-23K21403
[Journal Article] Congenital anaemia associated with loss-of-function variants in DNA polymerase epsilon 1.2024
- Author(s)
  Takeuchi I, Tanase-Nakao K, Ogawa A, Sugawara T, Migita O, Kashima M, Yamazaki T, Iguchi A, Naiki Y, Uchiyama T, Tamaoki J, Maeda H, Shimizu H, Kawai T, Taniguchi K, Hirata H, Kobayashi M, Matsumoto K, Naruse K, Hata K, Akutsu H, Kato T, Narumi S, Arai K, Ishiguro A.
- Journal Title
  
  J Med Genet.
  
  Volume: 61(3) Issue: 3 Pages: 239-243
- DOI
  10.1136/jmg-2023-109444
- Peer Reviewed / Open Access / Int'l Joint Research
- Data Source
  KAKENHI-PROJECT-22K07951, KAKENHI-PROJECT-19H03329, KAKENHI-PUBLICLY-22H05556, KAKENHI-PROJECT-20H00550
[Journal Article] A case of MYH7 and MYH9 genes variants with cardiomyopathy and macrothrombocytopenia.2024
- Author(s)
  Ikawa Y, Nakamura T, Fujino N, Uchiyama T, Ishiguro A, Takenaka M, Sakai Y, Noguchi K, Fujiki T, Wada T.
- Journal Title
  
  Clin Case Rep.
  
  Volume: 12(2) Issue: 2
- DOI
  10.1002/ccr3.8304
- Peer Reviewed / Open Access
- Data Source
  KAKENHI-PROJECT-22K07951, KAKENHI-PROJECT-20H03927
[Journal Article] Hematopoietic stem cell transplantation in two sisters with bone marrow failure associated with POLE gene variants.2024
- Author(s)
  Fujimori K, Ikenobe N, Gocho Y, Uchiyama T, Deguchi T, Sakaguchi H, Tomizawa D, Takeuchi I, Shimizu H, Arai K, Ishiguro A, Matsumoto K, Iguchi A.
- Journal Title
  
  Pediatr Blood Cancer.
  
  Volume: 71(5) Issue: 5
- DOI
  10.1002/pbc.30919
- Peer Reviewed / Open Access
- Data Source
  KAKENHI-PROJECT-22K07951
[Journal Article] Pediatric Hemophagocytic lymphohistiocytosis after concomitant administration of SARS-CoV-2 vaccine and influenza vaccine.2024
- Author(s)
  Kaizuka A, Tokuda Y, Morooka S, Gocho Y, Funaki T, Uchiyama T, Hirai Y, Yasumi T, Maekawa T, Kubota M, Ishiguro A.
- Journal Title
  
  J infect Chemother.
  
  Volume: 30(1) Issue: 1 Pages: 67-70
- DOI
  10.1016/j.jiac.2023.08.015
- Peer Reviewed / Open Access
- Data Source
  KAKENHI-PROJECT-22K07951
[Journal Article] HLA-haploidentical T-cell receptor αβT/B-cell-depleted stem cell transplantation for Fanconi anemia.2024
- Author(s)
  Iguchi A, Uchiyama T, Fujimori K, Gocho Y, Sakaguchi H, Deguchi T, Tomizawa D, Imadome KI, Onodera M, Matsumoto K.
- Journal Title
  
  Int J Hematol.
  
  Volume: 119(3) Issue: 3 Pages: 334-337
- DOI
  10.1007/s12185-023-03703-x
- Peer Reviewed / Open Access
- Data Source
  KAKENHI-PROJECT-22K07951
[Journal Article] Peripheral immune system modulates Purkinje cell degeneration in Niemann-Pick disease tyepe C1.2023
- Author(s)
  Yasuda T, Uchiyama T, Watanabe N, Ito N, Nakabayashi K, Mochizuki H, Onodera M.
- Journal Title
  
  Life Scie Alliance.
  
  Volume: 6(9) Issue: 9 Pages: e202201881-e202201881
- DOI
  10.26508/lsa.202201881
- Peer Reviewed / Open Access
- Data Source
  KAKENHI-PROJECT-22K07951
[Journal Article] Inherited macrothrombocytopenia due to a novel splice donor site mutation in ITGB3.2023
- Author(s)
  Komeno Y, Uchiyama T, Kawano F, Kurihara Y, Kurokawa M, Ohara O, Kunishima S, Ishiguro A.
- Journal Title
  
  Ann Hematol.
  
  Volume: 102(10) Issue: 10 Pages: 2947-2949
- DOI
  10.1007/s00277-023-05387-z
- Peer Reviewed / Open Access
- Data Source
  KAKENHI-PROJECT-22K07951
[Journal Article] レトロウイルス、レンチウイルスベクター―特徴から臨床応用まで2023
- Author(s)
  内山　徹
- Journal Title
  
  医学のあゆみ
  
  Volume: Vol. 285 No5 Pages: 321-327
- Data Source
  KAKENHI-PROJECT-22K07951
[Journal Article] Myelodysplasia after clonal hematopoiesis with APOBEC3-mediated CYBB inactivation in retroviral gene therapy for X-CGD2023
- Author(s)
  Uchiyama T、Kawai T、Nakabayashi K、Nakazawa Y、Goto F、Okamura K、Nishimura T、Kato K、Watanabe N、Miura A、Yasuda T、Ando Y、Minegishi T、Edasawa K、Shimura M、Akiba Y、Sato-Otsubo A、Mizukami T、Kato M、Akashi K、Nunoi H、Onodera M
- Journal Title
  
  Molecular Therapy
  
  Volume: 31 Issue: 12 Pages: 3424-3440
- DOI
  10.1016/j.ymthe.2023.09.004
- Peer Reviewed / Open Access
- Data Source
  KAKENHI-PROJECT-22K07928, KAKENHI-PROJECT-22K07951, KAKENHI-PROJECT-23K24490
[Journal Article] 遺伝子治療開発研究ハンドブック　第1章 Ex vivo遺伝子治療, 第1節5. Genotoxicity.2023
- Author(s)
  内山　徹
- Journal Title
  
  日本遺伝子細胞治療学会編集, 株式会社エヌ・ティー・エス.
  
  Volume: ‐
- Data Source
  KAKENHI-PROJECT-22K07951
[Journal Article] 原発性免疫不全症候群　診療の手引き　第1章複合免疫不全 B アデノシン・デアミナーゼ欠損症2023
- Author(s)
  内山　徹
- Journal Title
  
  日本免疫不全・自己炎症学会編集, 診断と治療社
  
  Volume: -
- Data Source
  KAKENHI-PROJECT-22K07951
[Journal Article] ゲノム編集の最新技術と医薬品・遺伝子治療・農業・水産物・有用物質生産への活用　第３章　遺伝子治療におけるゲノム編集技術の活用と課題対策　第5節　ゲノム編集技術による遺伝子治療の開発2023
- Author(s)
  内山　徹
- Journal Title
  
  技術情報協会
  
  Volume: -
- Data Source
  KAKENHI-PROJECT-22K07951
[Journal Article] Safety and efficacy of elapegademase in patients with adenosine deaminase deficiency: A multicenter, open‐label, single‐arm, phase 3, and postmarketing clinical study2023
- Author(s)
  Onodera M, Uchiyama T, Ariga T, Yamada M, Miyamura T, Arizono H, and Morio T.
- Journal Title
  
  Immunity, Inflammation and Disease
  
  Volume: 11 Issue: 7
- DOI
  10.1002/iid3.917
- Peer Reviewed / Open Access
- Data Source
  KAKENHI-PROJECT-20K08152, KAKENHI-PROJECT-22K07951
[Journal Article] Measurement of immature platelet fraction is useful in the differential diagnosis of MYH9 disorders.2023
- Author(s)
  Kouno H, Kunishima S, Takebe J, Uchiyama T, Kaname T, Ishiguro A.
- Journal Title
  
  Int J Lab Hematol.
  
  Volume: 45(5) Issue: 5 Pages: 700-706
- DOI
  10.1111/ijlh.14123
- Peer Reviewed / Open Access
- Data Source
  KAKENHI-PROJECT-22K07951
[Journal Article] 造血幹細胞遺伝子治療―原発性免疫不全症2023
- Author(s)
  内山　徹
- Journal Title
  
  日本医師会雑誌
  
  Volume: 第152巻（1） Pages: 248-252
- Data Source
  KAKENHI-PROJECT-22K07951
[Journal Article] ETV6-related thrombocytopenia associated with a transient decrease in von Willebrand factor2021
- Author(s)
  Kanamaru Yuri、Uchiyama Toru、Kaname Tadashi、Yanagi Kumiko、Ohara Osamu、Kunishima Shinji、Ishiguro Akira
- Journal Title
  
  International Journal of Hematology
  
  Volume: - Issue: 2 Pages: 297-300
- DOI
  10.1007/s12185-021-03136-4
- Peer Reviewed
- Data Source
  KAKENHI-PROJECT-19K08312, KAKENHI-PROJECT-20K09916, KAKENHI-PROJECT-18K07863
[Journal Article] Autoimmune hemolytic anemia associated with Takenouchi-Kosaki syndrome.2021
- Author(s)
  Ishikawa K, Uchiyama T, Kaname T, Kawai T, Ishiguro A.
- Journal Title
  
  Pediatr Int.
  
  Volume: 63(12) Issue: 12 Pages: 1528-1530
- DOI
  10.1111/ped.14651
- Peer Reviewed
- Data Source
  KAKENHI-PROJECT-19K08312
[Journal Article] Severe Liver Disorder Following Liver Transplantation in STING-Associated Vasculopathy with Onset in Infancy2021
- Author(s)
  Ishikawa Takashi、Tamura Eiichiro、Kasahara Mureo、Uchida Hajime、Higuchi Masataka、Kobayashi Hisato、Shimizu Hirotaka、Ogawa Eiki、Yotani Nobuyuki、Irie Rie、Kosaki Rika、Kosaki Kenjiro、Uchiyama Toru、Onodera Masafumi、Kawai Toshinao
- Journal Title
  
  Journal of Clinical Immunology
  
  Volume: - Issue: 5 Pages: 967-974
- DOI
  10.1007/s10875-021-00977-w
- Peer Reviewed
- Data Source
  KAKENHI-PROJECT-19K08312
[Journal Article] Nonconditioned ADA-SCID gene therapy reveals ADA requirement in the hematopoietic system and clonal dominance of vector-marked clones.2021
- Author(s)
  Uchiyama T, Sirirat Takahashi S, Nakabayashi K, Okamura K, Edasawa K, Yamada M, Watanabe N, Mochizuki E, Yasuda T, Miura A, Kato K, Tomizawa D, Otsu M, ArigaT , Onodera M.
- Journal Title
  
  Molecular Therapy: Methods & Clinical Development
  
  Volume: 23 Pages: 424-433
- DOI
  10.1016/j.omtm.2021.10.003
- Peer Reviewed / Open Access
- Data Source
  KAKENHI-PROJECT-19K08312, KAKENHI-PROJECT-20K08152, KAKENHI-PROJECT-21K06143
[Journal Article] Copy-number analysis of Y-linked loci in young men with non-obstructive azoospermia: Implications for the rarity of early onset mosaic loss of chromosome Y.2020
- Author(s)
  Suzuki E, Kobori Y, Katsumi M, Ushijima K, Uchiyama T, Okada H, Miyado M and Fukami M.
- Journal Title
  
  Reprod Med Biol.
  
  Volume: 19 Issue: 2 Pages: 178-181
- DOI
  10.1002/rmb2.12321
- Peer Reviewed / Open Access
- Data Source
  KAKENHI-PROJECT-17K11194, KAKENHI-PROJECT-19K09682, KAKENHI-PLANNED-17H06428, KAKENHI-PROJECT-17K11206, KAKENHI-PROJECT-19K08312
[Journal Article] Direct assessment of a single-cell DNA using crudely purified live cells: a proof of concept for noninvasive prenatal definitive diagnosis.2020
- Author(s)
  Saito T, Ito Y, Samura O, Aoki H, Uchiyama T, Okamoto A, Hata K
- Journal Title
  
  J Mol Diagn.
  
  Volume: 22 Issue: 2 Pages: 132-140
- DOI
  10.1016/j.jmoldx.2019.10.006
- Peer Reviewed
- Data Source
  KAKENHI-PROJECT-19K08312
[Journal Article] Comparison of clonazepam and levetiracetam in children for prevention of busulfan-induced seizure in hematopoietic stem cell transplantation.2020
- Author(s)
  11.Tsujimoto SI, Shirai R, Utano T, Osumi T, Matsumoto K, Shioda Y, Kiyotani C, Uchiyama T, Deguchi T, Terashima K, Tomizawa D, Matsumoto K, Kato M.
- Journal Title
  
  Int J Hematol.
  
  Volume: 111 Issue: 3 Pages: 463-466
- DOI
  10.1007/s12185-019-02795-8
- Peer Reviewed
- Data Source
  KAKENHI-PROJECT-19K08312
[Journal Article] A Distinct Feature of T Cell Subpopulations in a Patient with CHARGE Syndrome and Omenn Syndrome2020
- Author(s)
  Uchiyama Toru、Kawakami Saori、Masuda Hiroshi、Yoshida Kazue、Niizeki Hironori、Mochizuki Emi、Edasawa Kaori、Ishiguro Akira、Onodera Masafumi
- Journal Title
  
  Journal of Clinical Immunology
  
  Volume: 41 Issue: 1 Pages: 233-237
- DOI
  10.1007/s10875-020-00875-7
- Peer Reviewed
- Data Source
  KAKENHI-PROJECT-19K08312
[Journal Article] High prevalence of SMARCB1 constitutional abnormalities including mosaicism in malignant rhabdoid tumors2020
- Author(s)
  Shirai Ryota、Osumi Tomoo、Terashima Keita、et al
- Journal Title
  
  European Journal of Human Genetics
  
  Volume: 未刊 Issue: 8 Pages: 26-26
- DOI
  10.1038/s41431-020-0614-z
- Peer Reviewed / Open Access
- Data Source
  KAKENHI-PROJECT-17H01592, KAKENHI-PROJECT-18K15230, KAKENHI-PROJECT-19K08312, KAKENHI-PROJECT-19K22608, KAKENHI-PROJECT-20H00528
[Journal Article] Bacillus Calmette-Gu?rin (BCG) Infections at High Frequency in Both AR-CGD and X-CGD Patients Following BCG Vaccination2020
- Author(s)
  Ishikawa Takashi、Okai Masashi、Mochizuki Emi、Uchiyama Toru、Onodera Masafumi、Kawai Toshinao
- Journal Title
  
  Clinical Infectious Diseases
  
  Volume: - Issue: 9 Pages: e2538-e2544
- DOI
  10.1093/cid/ciaa1049
- Peer Reviewed
- Data Source
  KAKENHI-PROJECT-19K08312, KAKENHI-PROJECT-18K07807
[Journal Article] Prospective Study of Allogeneic Hematopoietic Stem Cell Transplantation with Post-Transplantation Cyclophosphamide and Antithymocyte Globulin from HLA-Mismatched Related Donors for Nonmalignant Diseases2020
- Author(s)
  Osumi T、Yoshimura S、Sako M、Uchiyama T、Ishikawa T、Kawai T、Inoue E、Takimoto T、Takeuchi I、Yamada M、Sakamoto K、Yoshida K、Kimura Y、Matsukawa Y、Matsumoto K、Imadome K、Arai K、Deguchi T、Imai K、Yuza Y、Matsumoto K、Onodera M、Kanegane H、Tomizawa D、Kato M
- Journal Title
  
  Biology of Blood and Marrow Transplantation
  
  Volume: 26 Issue: 11 Pages: e286-e291
- DOI
  10.1016/j.bbmt.2020.08.008
- Peer Reviewed
- Data Source
  KAKENHI-PROJECT-19K08312, KAKENHI-PROJECT-17H01592, KAKENHI-PROJECT-18K07807, KAKENHI-PROJECT-18K07814
[Journal Article] Efficacy of Antithymocyte Globulin Treatment for Severe Centrilobular Injury Following Pediatric Liver Transplant: Clinical Significance of Monitoring Lymphocyte Subset2020
- Author(s)
  Uchida Hajime、Sakamoto Seisuke、Shimizu Seiichi、Takeda Masahiro、Yanagi Yusuke、Fukuda Akinari、Uchiyama Toru、Irie Rie、Kasahara Mureo
- Journal Title
  
  Experimental and Clinical Transplantation
  
  Volume: 18 Issue: 3 Pages: 325-333
- DOI
  10.6002/ect.2019.0387
- Peer Reviewed
- Data Source
  KAKENHI-PROJECT-19K08312
[Journal Article] Low prevalence of maternal microchimerism in peripheral blood of Japanese children with type 1 diabetes2020
- Author(s)
  Ushijima K.、Okuno M.、Ayabe T.、Kikuchi N.、Kawamura T.、Urakami T.、Yokota I.、Amemiya S.、Uchiyama T.、Kikuchi T.、Ogata T.、Sugihara S.、Fukami M.、The Japanese Study Group of Insulin Therapy for Childhood and Adolescent Diabetes
- Journal Title
  
  Diabetic Medicine
  
  Volume: 37 Issue: 12 Pages: 2131-2135
- DOI
  10.1111/dme.14221
- Peer Reviewed
- Data Source
  KAKENHI-PROJECT-19K08312
[Journal Article] Haploinsufficiency of A20 caused by a novel nonsense variant or entire deletion of TNFAIP3 is clinically distinct from Beh?et’s disease2019
- Author(s)
  Tsuchida Naomi、Kirino Yohei、Soejima Yutaro、Onodera Masafumi、Arai Katsuhiro、Tamura Eiichiro、Ishikawa Takashi、Kawai Toshinao、Uchiyama Toru、Nomura Shigeru、Kobayashi Daisuke、Taguri Masataka、Mitsuhashi Satomi、Mizuguchi Takeshi、Takata Atsushi、Miyake Noriko、Nakajima Hideaki、Miyatake Satoko、Matsumoto Naomichi
- Journal Title
  
  Arthritis Research & Therapy
  
  Volume: 21 Issue: 1 Pages: 137-137
- DOI
  10.1186/s13075-019-1928-5
- Peer Reviewed / Open Access
- Data Source
  KAKENHI-PROJECT-17H01539, KAKENHI-PROJECT-16H06254, KAKENHI-PROJECT-17K10080, KAKENHI-PROJECT-19H03700, KAKENHI-PROJECT-19K08312
[Journal Article] A prospective study of allogeneic transplantation from unrelated donors for chronic granulomatous disease with target busulfan-based reduced-intensity conditioning.2019
- Author(s)
  Osumi T, Tomizawa D, Kawai T, Sako M, Inoue E, Takimoto T, Tamura E, Uchiyama T, Imadome K, Taniguchi M, Shirai R, Yoshida M, Ando R, Tsumura Y, Fuji H, Matsumoto K, Shioda Y, Kiyotani C, Terashima K, Onodera M, Matsumoto K, Kato M
- Journal Title
  
  Bone Marrow Transplant.
  
  Volume: 54 Issue: 1 Pages: 168-172
- DOI
  10.1038/s41409-018-0271-9
- Peer Reviewed
- Data Source
  KAKENHI-PROJECT-18K07807, KAKENHI-PROJECT-19K08312
[Journal Article] Acute promyelocytic leukemia with a cryptic insertion of RARA into TBL1XR12019
- Author(s)
  Osumi Tomoo、Watanabe Akihiro、Okamura Kohji、Nakabayashi Kazuhiko、Yoshida Masanori、Tsujimoto Shin‐ichi、Uchiyama Meri、Takahashi Hiroyuki、Tomizawa Daisuke、Hata Kenichiro、Kiyokawa Nobutaka、Kato Motohiro
- Journal Title
  
  Genes, Chromosomes and Cancer
  
  Volume: 58 Issue: 11 Pages: 820-823
- DOI
  10.1002/gcc.22791
- Peer Reviewed
- Data Source
  KAKENHI-PROJECT-19K16762, KAKENHI-PROJECT-17H01588, KAKENHI-PROJECT-17H04234
[Journal Article] Diplotype analysis of NUDT15 variants and 6-mercaptopurine sensitivity in pediatric lymphoid neoplasms2018
- Author(s)
  Tsujimoto S, Osumi T, Uchiyama M, Shirai R, Moriyama T, Nishii R, Yamada Y, Kudo K, Sekiguchi M, Arakawa Y, Yoshida M, Uchiyama T, Terui K, Ito S, Koh K, Takita J, Ito E, Tomizawa D, Manabe A, Kiyokawa N, Yang JJ, Kato M.
- Journal Title
  
  Leukemia
  
  Volume: 32 Issue: 12 Pages: 2710-2714
- DOI
  10.1038/s41375-018-0190-1
- Peer Reviewed / Int'l Joint Research
- Data Source
  KAKENHI-PROJECT-17K10129, KAKENHI-PROJECT-18K07836, KAKENHI-PROJECT-18K19467, KAKENHI-PROJECT-18H04039, KAKENHI-PROJECT-17H04224, KAKENHI-PROJECT-17H04234
[Journal Article] ABO-incompatible liver transplantation for children under 2?years of age: A case report and a single-center review2018
- Author(s)
  Narumoto Soichi、Sakamoto Seisuke、Sasaki Kengo、Hirata Yoshihiro、Fukuda Akinari、Uchiyama Toru、Irie Rie、Yoshioka Takako、Kasahara Mureo
- Journal Title
  
  Pediatric Transplantation
  
  Volume: 23 Issue: 1
- DOI
  10.1111/petr.13308
- Peer Reviewed / Int'l Joint Research
- Data Source
  KAKENHI-PROJECT-17K08756, KAKENHI-PROJECT-16K21668, KAKENHI-PROJECT-19K08312
[Journal Article] Somatic MECOM mosaicism in a patient with congenital bone marrow failure without a radial abnormality2018
- Author(s)
  Osumi Tomoo、Tsujimoto Shin-ichi、Nakabayashi Kazuhiko、Taniguchi Maki、Shirai Ryota、Yoshida Masanori、Uchiyama Toru、Nagasawa Junko、Goyama Susumu、Yoshioka Takako、Tomizawa Daisuke、Kurokawa Mineo、Matsubara Yoichi、Kiyokawa Nobutaka、Matsumoto Kimikazu、Hata Kenichiro、Kato Motohiro
- Journal Title
  
  Pediatric Blood & Cancer
  
  Volume: 65 Issue: 6
- DOI
  10.1002/pbc.26959
- Peer Reviewed
- Data Source
  KAKENHI-PROJECT-17H04234, KAKENHI-PROJECT-16K15533, KAKENHI-PROJECT-15K09676
[Journal Article] Persistent Impairment of T-Cell Regeneration in a Patient with Activated PI3K δ Syndrome2017
- Author(s)
  Goto F, Uchiyama T, Nakazawa Y, Imai K, Kawai T, Onodera M
- Journal Title
  
  J Clin Immunol
  
  Volume: 37 Issue: 4 Pages: 347-350
- DOI
  10.1007/s10875-017-0393-7
- Peer Reviewed
- Data Source
  KAKENHI-PROJECT-15K09640, KAKENHI-PROJECT-15K09676
[Journal Article] Fecal calprotectin rise in chronic granulomatous disease.2017
- Author(s)
  Nakazawa Y, Kawai T, Arai K, Tamura E, Uchiyama T, Onodera M
- Journal Title
  
  J. Clin. Immunol.
  
  Volume: 37 Pages: 741-743
- Peer Reviewed
- Data Source
  KAKENHI-PROJECT-15K09676
[Journal Article] ingle cell-based vector tracing in patients with ADA-SCID treated with stem cell gene therapy.2017
- Author(s)
  Igarashi Y, Uchiyama T, Minegishi T, Takahashi S, Watanabe N, Kawai T, Yamada M, Ariga T, Onodera M
- Journal Title
  
  Mol. Ther. Methods. Clin. Dev
  
  Volume: 6 Pages: 8-16
- DOI
  10.1016/j.omtm.2017.05.005
- Peer Reviewed / Open Access
- Data Source
  KAKENHI-PROJECT-15K09676
[Journal Article] Blastic transformation of juvenile myelomonocytic leukemia caused by the copy number gain of oncogenic KRAS.2017
- Author(s)
  Osumi T, Kato M, Ouchi-Uchiyama M, Tomizawa D, Kataoka K, Fujii Y, Seki M, Takita J, Ogawa S, Uchiyama T, Ohki K, Kiyokawa N.
- Journal Title
  
  Pediatr Blood Cancer. Pediatr Blood Cancer.
  
  Volume: 64(9) Issue: 9
- DOI
  10.1002/pbc.26496
- Peer Reviewed / Open Access
- Data Source
  KAKENHI-PROJECT-17H04224, KAKENHI-PROJECT-17H04234, KAKENHI-PROJECT-15K09676
[Journal Article] A Cryptic NUP214-ABL1 Fusion in B-cell Precursor Acute Lymphoblastic Leukemia.2017
- Author(s)
  Tsujimoto SI, Nakano Y, Osumi T, Okada K, Ouchi-Uchiyama M, Kataoka K, Fujii Y, Ohki K, Seki M, Tamagawa N, Takita J, Ogawa S, Kiyokawa N, Hara J, Kato M.
- Journal Title
  
  J Pediatr Hematol Oncol.
  
  Volume: － Issue: 6 Pages: 1-1
- DOI
  10.1097/mph.0000000000001007
- Peer Reviewed / Open Access
- Data Source
  KAKENHI-PROJECT-17H04224, KAKENHI-PROJECT-17H04234
[Journal Article] The long terminal repeat negative control region is a critical element for insertion oncogenesis after gene transfer into hematopoietic progenys with Moloney murine leukemia viral vectors.2016
- Author(s)
  Ikawa Y, Uchiyama T, Jagadeesh GJ, Candotti F
- Journal Title
  
  Gene therapy
  
  Volume: 23 Issue: 11 Pages: 815-818
- DOI
  10.1038/gt.2016.51
- Peer Reviewed / Acknowledgement Compliant / Int'l Joint Research
- Data Source
  KAKENHI-PROJECT-15K09676
[Journal Article] Effects of enzyme replacement therapy on immune function in ADA deficiency patient2015
- Author(s)
  Nakazawa Y, Kawai T, Uchiyama T, Goto F, Watanabe N, Maekawa T, Ishigro A, Okuyama, T, Otsu M, Yamada M, Hershfield MS, Ariga T, Onodera M
- Journal Title
  
  Clinical Immunology
  
  Volume: 161 Issue: 2 Pages: 391-393
- DOI
  10.1016/j.clim.2015.06.011
- Peer Reviewed / Acknowledgement Compliant / Int'l Joint Research
- Data Source
  KAKENHI-PROJECT-15K09676
[Journal Article] Severe and Rapid Progression in Very Early-Onset Chronic Granulomatous Disease-Associated Colitis.2015
- Author(s)
  Kawai T, Arai K, Harayama S, Nakazawa Y, Goto F, Maekawa T, Tamura E, Uchiyama T, Onodera M
- Journal Title
  
  Journal if Clinical Immunology
  
  Volume: 35 Issue: 6 Pages: 583-588
- DOI
  10.1007/s10875-015-0180-2
- Peer Reviewed / Acknowledgement Compliant / Open Access / Int'l Joint Research
- Data Source
  KAKENHI-PROJECT-15K09676, KAKENHI-PROJECT-15K09677
[Journal Article] 原発性免疫不全－複合免疫不全2014
- Author(s)
  内山徹
- Journal Title
  
  小児の治療指針
  
  Volume: 77 Pages: 228-230
- Data Source
  KAKENHI-PROJECT-25860836
[Journal Article] Interstitial Lung Disease with Multiple Microgranulomas in Chronic Granulomatous Disease.2014
- Author(s)
  Kawai T, Watanabe N, Yokoyama M, Nakazawa Y, Goto F, Uchiyama T, Higuchi M, Maekawa T, Tamura E, Nagasaka S, Hojo M, Onodera M
- Journal Title
  
  Journal of Clinical Immunology
  
  Volume: 34 Issue: 8 Pages: 933-940
- DOI
  10.1007/s10875-014-0089-1
- Peer Reviewed / Acknowledgement Compliant
- Data Source
  KAKENHI-PROJECT-25860836
[Journal Article] The open conformation of WASP regulates its nuclear localization and gene transcription in myeloid cells.2014
- Author(s)
  2.Looi CY, Sasahara Y, Watanabe Y, Sato M, Hakozaki I, Uchiyama M, Wong WF, Uchiyama T, Kumaki S, Tsuchiya S, Kure S
- Journal Title
  
  International Immnology
  
  Volume: 26 Issue: 6 Pages: 341-352
- DOI
  10.1093/intimm/dxt072
- Peer Reviewed / Acknowledgement Compliant / Open Access
- Data Source
  KAKENHI-PROJECT-25860836, KAKENHI-PROJECT-26461562, KAKENHI-PROJECT-26461597, KAKENHI-PROJECT-26860780
[Journal Article] A case series of CAEBV of children and young adults treated with reduced-intensity conditioning and allogeneic bone marrow transplantation: a single-center study.2013
- Author(s)
  Watanabe Y, Sasahara Y, Satoh M, Looi CY, Katayama S, Suzuki T, Suzuki N, Ouchi M, Horino S, Moriya K, Nanjyo Y, Onuma M, Kitazawa H, Irie M, Niizuma H, Uchiyama T, Rikiishi T, Kumaki S, Minegishi M, Wada T, Yachie A, Tsuchiya S, Kure S.
- Journal Title
  
  Eur J Haematol
  
  Volume: 91 Issue: 3 Pages: 242-248
- DOI
  10.1111/ejh.12151
- Peer Reviewed
- Data Source
  KAKENHI-PROJECT-24591542, KAKENHI-PROJECT-25860836
[Journal Article] Interstitial lung disease in two brothers with novel compound heterozygous ABCA3 mutations2013
- Author(s)
  Kitazawa H, Moriya K, Niizuma H, Kawano K, Saito-Nanjo Y, Uchiyama T, Rikiishi T, Sasahara Y, Sakamoto O, Setoguchi Y, Kure S
- Journal Title
  
  Eur J Pediatr
  
  Volume: 172(7) Issue: 7 Pages: 953-7
- DOI
  10.1007/s00431-013-1977-8
- Peer Reviewed
- Data Source
  KAKENHI-PROJECT-23390268, KAKENHI-PROJECT-23791147, KAKENHI-PROJECT-25461532, KAKENHI-PROJECT-25860836
[Journal Article] Current status and future prospects of stem cell gene therapy for primary immunodeficiency2013
- Author(s)
  Uchiyama T, Onodera M
- Journal Title
  
  Nihon Rinsho Meneki Gakkai kaishi
  
  Volume: 36 Pages: 148-155
- NAID
  10031185521
- Data Source
  KAKENHI-PROJECT-25860836
[Journal Article] Gene therapy model of X-linked severe combined immunodeficiency using a modified foamy virus vector2013
- Author(s)
  Horino S, Uchiyama T, So T, Nagashima H, Sun SL, Sato M, Asao A, Haji Y, Sasahara Y, Candotti F, Tsuchiya S, Kure S, Sugamura K, and Ishii N
- Journal Title
  
  PLoS ONE
  
  Volume: 8 Issue: 8 Pages: e71594-e71594
- DOI
  10.1371/journal.pone.0071594
- Peer Reviewed
- Data Source
  KAKENHI-PROJECT-11J06848, KAKENHI-PROJECT-24659486, KAKENHI-PROJECT-24659487, KAKENHI-PROJECT-25290047, KAKENHI-PROJECT-25860836
[Journal Article] Foamy Virus Vector-mediated Gene Correction of a Mouse Model of Wiskott-Aldrich Syndrome2012
- Author(s)
  Uchiyama T, Adriani M, Jagadeesh GJ, Paine A, Candotti F
- Journal Title
  
  Mol. Ther
  
  Volume: 20(6) Issue: 6 Pages: 1270-1279
- DOI
  10.1038/mt.2011.282
- Peer Reviewed
- Data Source
  KAKENHI-PROJECT-23659511, KAKENHI-PROJECT-23791147
[Journal Article] Development of a Multi-Step Leukemogenesis Model of MLL-Rearranged Leukemia Using Humanized Mice2012
- Author(s)
  Moriya K, Suzuki M, Watanabe Y, Takahashi T, Aoki Y, Uchiyama T, Kumaki S, Sasahara Y, Minegishi M, Kure S, Tsuchiya S, Sugamuta K, Ishii N
- Journal Title
  
  PLos One
  
  Volume: 7(6)
- Peer Reviewed
- Data Source
  KAKENHI-PROJECT-23659511
[Journal Article] Successful cord blood transplantation with reduced-intensity conditioning for childhood cerebral X-linked adrenoleukodystrophy at advanced and early stages2012
- Author(s)
  Niizuma H, Uematsu M, Sakamoto O, Uchiyama T, Horino S, Onuma M, Matsuhashi T, Rikiishi T, Sasahara Y, Minegishi M, Tsuchiya S.
- Journal Title
  
  Pediatr Transplant
  
  Volume: 16(2) Issue: 2
- DOI
  10.1111/j.1399-3046.2011.01539.x
- Peer Reviewed
- Data Source
  KAKENHI-PROJECT-22591150, KAKENHI-PROJECT-23659511, KAKENHI-PROJECT-23791147
[Journal Article] Development of a Multi-Step Leukemogenesis Model of MLL-Rearranged Leukemia Using Humanized Mice2012
- Author(s)
  Moriya K, Suzuki M, Watanabe Y, Takahashi T, Aoki Y, Uchiyama T, Kumaki S, Sasahara Y, Minegishi M, Kure S, Tsuchiya S, Sugamuta K, Ishii N.
- Journal Title
  
  PLos One
  
  Volume: 7(6)
- Peer Reviewed
- Data Source
  KAKENHI-PROJECT-23791147
[Journal Article] Defective inhibition of B-cell proliferation by Wiskott-Aldrich syndrome protein-deficient regulatory T cells2011
- Author(s)
  Adriani M, Jones KA, Uchiyama T, Kirby MR, Silvin C, Anderson SM, Candotti F
- Journal Title
  
  Blood
  
  Volume: 117(24) Issue: 24 Pages: 6608-6611
- DOI
  10.1182/blood-2010-12-322834
- Peer Reviewed
- Data Source
  KAKENHI-PROJECT-23659511, KAKENHI-PROJECT-23791147
[Journal Article] Successful cord blood transplantation with reduced-intensity conditioning for childhood cerebral X-linked adrenoleukodystorophy at advanced and early stages
- Author(s)
  Niizuma H, Uematsu M, Sakamoto O, Uchiyama T, Horino S, Onuma M, Matsuhashi T, Rikiishi T, Sasahara Y, Minegishi M, Tsuchiya S.
- Journal Title
  
  Pediatr. Transplant
  
  Volume: 16(2)
- Peer Reviewed
- Data Source
  KAKENHI-PROJECT-23791147
[Journal Article] Interstitial lung disease in two brothers with novel compound heterozygous ABCA3 mutations
- Author(s)
  Kitazawa H., Moriya K., Niizuma H., Kawano K., Saito-Nanjo Y., Uchiyama T., Rikiishi T., Sasahara Y., Sakamoto O., Setoguchi Y., Kure S
- Journal Title
  
  Eur J Pediatr
  
  Volume: (In press)
- Peer Reviewed
- Data Source
  KAKENHI-PROJECT-23791147
[Presentation] A sc-ddPCR vector tracing in ADA-SCID stem cell gene therapy.2023
- Author(s)
  Toru Uchiyama,
- Organizer
  Bio-Rad Droplet Digital PCR World 2023.
- Invited / Int'l Joint Research
- Data Source
  KAKENHI-PROJECT-22K07951
[Presentation] Development of gene and cell therapy for intractable diseases in childhood.2023
- Author(s)
  内山　徹
- Organizer
  第29回日本遺伝子細胞治療学会学術集会　シンポジウム
- Invited
- Data Source
  KAKENHI-PROJECT-22K07951
[Presentation] 小児希少疾患に対する遺伝子治療2023
- Author(s)
  内山　徹
- Organizer
  第46回日本小児遺伝学会学術集会　特別企画シンポジウム
- Invited
- Data Source
  KAKENHI-PROJECT-22K07951
[Presentation] Gene-based monitoring in gene therapy for intractable diseases in Childhood.2023
- Author(s)
  Toru Uchiyama,
- Organizer
  Bio-Rad Droplet Digital PCR World 2023.
- Invited / Int'l Joint Research
- Data Source
  KAKENHI-PROJECT-22K07951
[Presentation] Gene therapy for inborn errors of immunity.2023
- Author(s)
  内山　徹
- Organizer
  第29回日本遺伝子細胞治療学会学術集会　シンポジウム
- Invited
- Data Source
  KAKENHI-PROJECT-22K07951
[Presentation] 新生児スクリーニングにて発見され、迅速に酵素補充療法を導入したアデノシン・デアミナーゼ欠損症の一例2022
- Author(s)
  内山徹、岡井真史、坂口大俊、坂本淳、馬場みのり、田中英、入月浩美、長崎啓佑、石川尊士、井口晶裕、河合利尚、今井千速、富澤大輔、小野寺雅史
- Organizer
  第5回日本免疫不全・自己炎症学会総会・学術集会
- Data Source
  KAKENHI-PROJECT-19K08312
[Presentation] アデノシン・デアミナーゼ欠損症における酵素補充療法による免疫再構築の評価,2022
- Author(s)
  内山徹、山田雅文、宮村能子、小野寺雅史
- Organizer
  第5回日本免疫不全・自己炎症学会総会・学術集会
- Data Source
  KAKENHI-PROJECT-19K08312
[Presentation] Progress of gene therapy for primary immunodeficiency diseases.2021
- Author(s)
  内山徹
- Organizer
  第27回日本遺伝子細胞治療学会学術集会
- Data Source
  KAKENHI-PROJECT-19K08312
[Presentation] CAR-T細胞の体内動態と有効性、安全性の検討2021
- Author(s)
  内山　徹
- Organizer
  第11回　国際協力遺伝病遺伝子治療フォーラム
- Invited
- Data Source
  KAKENHI-PROJECT-19K08312
[Presentation] 原発性免疫不全症へのゲノム編集による遺伝子治療法の開発2021
- Author(s)
  内山徹
- Organizer
  日本ゲノム編集学会第６回大会
- Data Source
  KAKENHI-PROJECT-19K08312
[Presentation] Progress in the treatment of primary immunodeficiency -enzyme replacement therapy and gene therapy-2020
- Author(s)
  内山　徹
- Organizer
  第3回日本免疫不全・自己炎症学会
- Invited
- Data Source
  KAKENHI-PROJECT-19K08312
[Presentation] 再生医療　-遺伝子治療と細胞治療-2020
- Author(s)
  内山　徹
- Organizer
  第20回CRCと臨床試験のあり方を考える会議2020 in長崎
- Invited
- Data Source
  KAKENHI-PROJECT-19K08312
[Presentation] Progress of gene therapy for primary immunodeficiency diseases.2020
- Author(s)
  内山　徹
- Organizer
  第10回国際協力遺伝病遺伝子治療フォーラム第10周年記念大会
- Invited
- Data Source
  KAKENHI-PROJECT-19K08312
[Presentation] レンチウイルスベクターの品質・安全管理2020
- Author(s)
  内山　徹
- Organizer
  第20回医薬品等ウイルス安全性シンポジウム
- Invited
- Data Source
  KAKENHI-PROJECT-19K08312
[Presentation] Progress of gene therapy for primary immunodeficiency.2019
- Author(s)
  内山　徹
- Organizer
  第25回日本遺伝子細胞治療学会
- Invited
- Data Source
  KAKENHI-PROJECT-19K08312
[Presentation] Insertional Oncogenesis in X-CGD Patient after MFGS Retroviral Vector-Mediated Gene Therapy.2019
- Author(s)
  Toru Uchiyama
- Organizer
  22nd Annual meeting of American Society of Gene and Cell therapy.
- Int'l Joint Research
- Data Source
  KAKENHI-PROJECT-19K08312
[Presentation] X連鎖慢性肉芽腫症へのレトロウイルスベクターによる遺伝子治療における発がん挿入変異の解析2019
- Author(s)
  内山　徹
- Organizer
  第81回日本血液学会学術集会
- Data Source
  KAKENHI-PROJECT-19K08312
[Presentation] Single cell-based vector tracing in patients with ADA-SCID treated with stem cell gene therapy.2019
- Author(s)
  Toru Uchiyama
- Organizer
  2019 Cell Therapy Seminar
- Invited / Int'l Joint Research
- Data Source
  KAKENHI-PROJECT-19K08312
[Presentation] Current status of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome in Japan2017
- Author(s)
  Uchiyama T
- Organizer
  The 7th International Collaboration Forum of Human Gene Therapy for Genetic Disease
- Place of Presentation
  東京
- Invited
- Data Source
  KAKENHI-PROJECT-15K09676
[Presentation] Current status of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome in Japan.2017
- Author(s)
  Toru Uchiyama
- Organizer
  The 7th International Collaboration Forum of Human Gene Therapy for Genetic Disease.
- Invited
- Data Source
  KAKENHI-PROJECT-15K09676
[Presentation] Progress in gene therapy for primary immunodeficiencies2017
- Author(s)
  内山　徹
- Organizer
  第23回日本遺伝子細胞治療学会
- Invited
- Data Source
  KAKENHI-PROJECT-15K09676
[Presentation] デノシンデアミナーゼ欠損症の従姉弟例2017
- Author(s)
  内山　徹
- Organizer
  第10回日本免疫不全症研究会
- Data Source
  KAKENHI-PROJECT-15K09676
[Presentation] Single cell-based vector tracing in the patients treated with stem cell gene therapy2016
- Author(s)
  Takahashi S, Igarashi Y, Uchiyama T, Onodera M.
- Organizer
  第22回日本遺伝子治療学会学術集会
- Place of Presentation
  東京
- Year and Date
  2016-07-28
- Data Source
  KAKENHI-PROJECT-15K09676
[Presentation] Selective pressure of vector-derived ADA in ADA-SCID patients treated with gene therapy2016
- Author(s)
  内山徹、五十嵐友香、渡辺信之、高橋シリラット、中澤裕美子、河合利尚、後藤文洋、山田雅文、有賀正、小野寺雅史
- Organizer
  第78回日本血液学会学術集会
- Place of Presentation
  横浜
- Year and Date
  2016-10-13
- Data Source
  KAKENHI-PROJECT-15K09676
[Presentation] Hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome2016
- Author(s)
  Toru Uchiyama, Satoshi Horino, Tkanori So, G Jayashree Jagadeesh, Naoto Ishii, Fabio Candotti
- Organizer
  Japan Society of Gene and Cell Therapy, 22nd Annual Meeting
- Place of Presentation
  Tokyo
- Year and Date
  2016-07-28
- Invited
- Data Source
  KAKENHI-PROJECT-15K09676
[Presentation] 乾燥ろ紙血を用いた原発性免疫不全症の新生児マススクリーニングパイロット研究2016
- Author(s)
  河合利尚、内山徹、後藤文洋、中澤裕美子、小須賀基通、和田友香、塚本桂子、伊藤裕司、奥山虎之、小野寺雅史
- Organizer
  第119回小児科学会学術集会
- Place of Presentation
  札幌
- Year and Date
  2016-05-13
- Data Source
  KAKENHI-PROJECT-15K09676
[Presentation] 重症複合免疫不全症（SCID）の新生児スクリーニング2016
- Author(s)
  後藤文洋、長田香代、峰岸知子、諸田沙織、中島英規、奥山虎之、河合利尚、小野寺雅史、内山徹
- Organizer
  第４３回日本マススクリーニング学会
- Place of Presentation
  札幌
- Year and Date
  2016-08-26
- Data Source
  KAKENHI-PROJECT-15K09676
[Presentation] Hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome2016
- Author(s)
  Uchiyama T
- Organizer
  第22回日本遺伝子治療学会学術集会
- Place of Presentation
  東京
- Year and Date
  2016-07-28
- Invited
- Data Source
  KAKENHI-PROJECT-15K09676
[Presentation] A novel tracking system of transgene using droplet-based single cell PCR in hematopoietic stem cell gene therapy2015
- Author(s)
  Igarashi Y, Uchiyama T, Wanatabe N, Nakazawa Y, Otsu M, Ariga T, Onodera M
- Organizer
  European Society of Gene and Cell Therapy and Finnish Society of Gene therapy Collaborative Congress
- Place of Presentation
  Helsinki, Finland
- Year and Date
  2015-09-17
- Int'l Joint Research
- Data Source
  KAKENHI-PROJECT-15K09676
[Presentation] Influence of virus production methods on genotoxic potential of gene transfer vectors2015
- Author(s)
  Takahashi S, Uchiyama T, Onodera M
- Organizer
  Japan Society of Gene Therapy, 21th Annual Meeting
- Place of Presentation
  Osaka
- Year and Date
  2015-07-24
- Data Source
  KAKENHI-PROJECT-15K09676
[Presentation] Regulatory science of viral vector for gene therapy2015
- Author(s)
  Igarashi Y, Uchiyama T, Uchida E, Onodera M
- Organizer
  Japan Society of Gene Therapy, 21th Annual Meeting
- Place of Presentation
  Osaka
- Year and Date
  2015-07-24
- Data Source
  KAKENHI-PROJECT-15K09676
[Presentation] Rapamycin controls lymph proliferation and restores functional T cells in Activated PI3Kd syndrome2015
- Author(s)
  Uchiyama T
- Organizer
  第77回日本血液学会学術集会
- Place of Presentation
  金沢
- Year and Date
  2015-10-16
- Data Source
  KAKENHI-PROJECT-15K09676
[Presentation] 原発性免疫不全症に対する造血幹細胞移植2015
- Author(s)
  内山徹
- Organizer
  第５回国際遺伝病遺伝子治療フォーラム
- Place of Presentation
  東京
- Year and Date
  2015-01-15
- Invited
- Data Source
  KAKENHI-PROJECT-25860836
[Presentation] 原発性免疫不全症への造血幹細胞遺伝子治療の現況2014
- Author(s)
  内山　徹
- Organizer
  第４回関西免疫不全症研究会
- Place of Presentation
  大阪
- Year and Date
  2014-07-19
- Invited
- Data Source
  KAKENHI-PROJECT-25860836
[Presentation] Effects of chronic granulomatous disease-associated bowel inflammation on microbial gut flora.2013
- Author(s)
  Kawai T, Nakazawa Y, Uchiyama T, Arai K. Onodera M
- Organizer
  第13回アジア汎太平洋小児栄養消化器肝臓学会
- Place of Presentation
  東京
- Data Source
  KAKENHI-PROJECT-25860836
[Presentation] 単球の活性酸素産生能低下による肉芽腫性腸炎2013
- Author(s)
  河合利尚、中澤裕美子、田村英一郎、清水泰岳、伊藤玲子、内山徹
- Organizer
  第45回日本小児感染症学会
- Place of Presentation
  札幌
- Data Source
  KAKENHI-PROJECT-25860836
[Presentation] Effect of Thalidomide Therapy on Bowel Inflammation in Chronic Granulomatous Disease.2013
- Author(s)
  Nakazawa Y, Kawai T, Uchiyama T, Arai K. Onodera M
- Organizer
  第13回アジア汎太平洋小児栄養消化器肝臓学会
- Place of Presentation
  東京
- Data Source
  KAKENHI-PROJECT-25860836
[Presentation] Gene Therapy models of X-linked severe combined immunodeficiency and Wiskott-Aldrich syndrome using foamy virus vectors.2013
- Author(s)
  Toru Uchiyama, Satoshi Horino, Tkanori So, G Jayashree Jagadeesh, Naoto Ishii, Fabio Candotti
- Organizer
  European Society of Gene and Cell Therapy Congress 2013
- Place of Presentation
  Madrid, Spain
- Data Source
  KAKENHI-PROJECT-25860836
[Presentation] 慢性肉芽腫症における肺間質性炎症2013
- Author(s)
  河合利尚、中澤裕美子、渡辺信之、横山みどり、内山徹、田村英一郎、伊藤秀一、放生雅章、小野寺雅史
- Organizer
  第23回日本小児リウマチ学会
- Place of Presentation
  埼玉
- Data Source
  KAKENHI-PROJECT-25860836
[Presentation] Spontaneous reversionを伴う原発性免疫不全症2症例における長期的臨床経過の検討2012
- Author(s)
  笹原洋二、佐藤美季、斉藤由佳、小沼正栄、入江正寛、内山徹、力石健、呉繁夫
- Organizer
  第115回日本小児科学会学術集会
- Place of Presentation
  福岡
- Year and Date
  2012-04-20
- Data Source
  KAKENHI-PROJECT-23659511
[Presentation] リツキシマブが有効であった乳児自己免疫性溶血性貧血の1例2012
- Author(s)
  森谷邦彦、松橋徹朗、小沼正栄、内山徹、力石健、浅田洋司、鈴木潤、笹原洋二、呉繁夫
- Organizer
  第54回小児血液・がん学会
- Place of Presentation
  横浜
- Year and Date
  2012-11-30
- Data Source
  KAKENHI-PROJECT-23659511
[Presentation] Spontaneous reversionを伴う原発性免疫不全症2症例における長期的臨床経過の検討2012
- Author(s)
  笹原洋二、佐藤美季、斉藤由佳、小沼正栄、入江正寛、内山徹、力石健、呉繁夫
- Organizer
  第115回日本小児科学会学術集会
- Place of Presentation
  福岡
- Year and Date
  2012-04-20
- Data Source
  KAKENHI-PROJECT-23791147
[Presentation] 骨髄非破壊的前処置にて同種骨髄移植を施行した慢性活動性EBV感染症4例の臨床的検討2012
- Author(s)
  笹原洋二、渡辺祐子、小沼正栄、新妻秀剛、内山徹、力石健、峯岸政好、久間木悟、土屋滋
- Organizer
  第34回日本造血幹細胞移植学会学術集会
- Place of Presentation
  大阪
- Year and Date
  2012-02-24
- Data Source
  KAKENHI-PROJECT-23659511
[Presentation] Development of Foamy virus vectors for primary immunodeficiencies.2012
- Author(s)
  内山徹
- Organizer
  第18回日本遺伝子治療学会
- Place of Presentation
  熊本
- Data Source
  KAKENHI-PROJECT-23791147
[Presentation] RISTを施行した当科年長児および成人期WAS/XLT3症例の臨床的検討2012
- Author(s)
  笹原洋二、力石健、北沢博、内山徹、森尾友宏、峯岸政好、久間木悟、土屋滋
- Organizer
  第5回日本免疫不全症研究会
- Place of Presentation
  東京
- Year and Date
  2012-01-21
- Data Source
  KAKENHI-PROJECT-23659511
[Presentation] Fabio Candotti Development of Foamy Virus Vector System for Primaryimmunodeficiencies2012
- Author(s)
  Toru Uchiyama、Satoshi Horino、Yoji Sasahara、Naoto Ishii
- Organizer
  第18回日本遺伝子治療学会
- Place of Presentation
  熊本
- Year and Date
  2012-06-28
- Data Source
  KAKENHI-PROJECT-23659511
[Presentation] Development of Foamy Virus Vector System for Primaryimmunodeficiencies2012
- Author(s)
  Toru Uchiyama 、Satoshi Horino 、Yoji Sasahara、Naoto Ishii、Fabio Candotti
- Organizer
  第18回日本遺伝子治療学会
- Place of Presentation
  熊本
- Year and Date
  2012-06-28
- Data Source
  KAKENHI-PROJECT-23791147
[Presentation] 骨髄非破壊的前処置にて同種骨髄移植を施行した慢性活動性EBV感染症4例の臨床的検討2012
- Author(s)
  笹原洋二、渡辺祐子、小沼正栄、新妻秀剛、内山徹、力石健、峯岸政好、久間木悟、土屋滋
- Organizer
  第34回日本造血幹細胞移植学会学術集会
- Place of Presentation
  大阪
- Year and Date
  2012-02-24
- Data Source
  KAKENHI-PROJECT-23791147
[Presentation] RISTを施行した当科年長児および成人期WAS/XLT3症例の臨床的検討2012
- Author(s)
  笹原洋二、力石健、北沢博、内山徹、森尾友宏、峯岸政好、久間木悟、土屋滋
- Organizer
  第5回日本免疫不全症研究会
- Place of Presentation
  東京
- Year and Date
  2012-01-21
- Data Source
  KAKENHI-PROJECT-23791147
[Presentation] リツキシマブが有効であった乳児自己免疫性溶血性貧血の1例2012
- Author(s)
  森谷邦彦、松橋徹朗、小沼正栄、内山徹、力石健、浅田洋司、鈴木潤、笹原洋二、呉繁夫
- Organizer
  第54回小児血液・がん学会学術集会
- Place of Presentation
  横浜
- Data Source
  KAKENHI-PROJECT-23791147
[Presentation] SKY法が複雑型染色体異常の同定に有用であった急性白血病の2例2011
- Author(s)
  片山紗乙莉、小沼正栄、内山徹、新妻秀剛、力石健、笹原洋二、呉繁夫
- Organizer
  第53回日本小児血液・がん学会学術集会
- Place of Presentation
  前橋
- Year and Date
  2011-11-25
- Data Source
  KAKENHI-PROJECT-23659511
[Presentation] SKY法が複雑型染色体異常の同定に有用であった急性白血病の2例2011
- Author(s)
  片山紗乙莉、小沼正栄、内山徹、新妻秀剛、力石健、笹原洋二、呉繁夫
- Organizer
  第53回日本小児血液・がん学会学術集会
- Place of Presentation
  前橋
- Year and Date
  2011-11-25
- Data Source
  KAKENHI-PROJECT-23791147
[Presentation] The progress of stem cell gene therapy for primary immunodefici- ency2011
- Author(s)
  Toru Uchiyama
- Organizer
  第17回日本遺伝子治療学会学術集会、シンポジウム
- Place of Presentation
  福岡
- Year and Date
  2011-07-15
- Data Source
  KAKENHI-PROJECT-23791147
[Presentation] フォーミーウイルスベクターによるWiskott-Aldrich症候群への遺伝子治療2011
- Author(s)
  内山徹、Fabio Candotti、土屋滋
- Organizer
  第114回日本小児科学会学術集会
- Place of Presentation
  東京
- Year and Date
  2011-08-12
- Data Source
  KAKENHI-PROJECT-23659511
[Presentation] The progress of stem cell gene therapy for primary immunodeficiency2011
- Author(s)
  Toru Uchiyama
- Organizer
  第17回日本遺伝子治療学会学術集会
- Place of Presentation
  福岡
- Year and Date
  2011-07-15
- Data Source
  KAKENHI-PROJECT-23659511
[Presentation] フォーミーウイルスベクターによるWiskott-Aldrich症候群への遺伝子治療2011
- Author(s)
  内山徹、Fabio Candotti、土屋滋
- Organizer
  第114回日本小児科学会学術集会
- Place of Presentation
  東京
- Year and Date
  2011-08-12
- Data Source
  KAKENHI-PROJECT-23791147
[Presentation] Foamy virus vectorを用いたX連鎖重症複合免疫不全症に対する遺伝子治療モデルの作製
- Author(s)
  堀野智史、内山徹、宗孝紀、佐藤美季、孫舒嵐、笹原洋二、土屋滋、呉繁夫、菅村和夫、石井直人
- Organizer
  第117回小児科学会学術集会
- Place of Presentation
  名古屋
- Year and Date
  2014-04-11 – 2014-04-13
- Data Source
  KAKENHI-PROJECT-25860836
[Presentation] 慢性肉芽腫症の炎症性肉芽腫に対するサリドマイドの治療効果
- Author(s)
  河合利尚、中澤裕美子、後藤文洋、内山徹、新井勝大、久保田雅也、石黒精、布井博幸、小林正夫、小野寺雅史
- Organizer
  第117回小児科学会学術集会
- Place of Presentation
  名古屋
- Year and Date
  2014-04-11 – 2014-04-13
- Data Source
  KAKENHI-PROJECT-25860836
[Presentation] 慢性肉芽腫症42例における肉芽腫性疾患の臨床的検討
- Author(s)
  後藤文洋、河合利尚、中澤裕美子、内山徹、原山静子、田村英一郎、亀井宏一、新井勝大、小野寺雅史
- Organizer
  第117回小児科学会学術集会
- Place of Presentation
  名古屋
- Year and Date
  2014-04-11 – 2014-04-13
- Data Source
  KAKENHI-PROJECT-25860836
[Presentation] 酸素補充療法が造血幹細胞遺伝子治療後のADA欠損症患者に与える免疫機能改善効果
- Author(s)
  中澤裕美子、河合利尚、後藤文洋、内山徹、渡辺信之、前川貴伸、石黒精、奥山虎之、山田雅文、大津真、有賀正、小野寺雅史
- Organizer
  第117回小児科学会学術集会
- Place of Presentation
  名古屋
- Year and Date
  2014-04-11 – 2014-04-13
- Data Source
  KAKENHI-PROJECT-25860836

1. Kato Motohiro (40708690)

# of Collaborated Projects: 2 results

# of Collaborated Products: 1 results
2. 中林一彦 (10415557)

# of Collaborated Projects: 2 results

# of Collaborated Products: 1 results
3. 内田直也 (70866821)

# of Collaborated Projects: 2 results

# of Collaborated Products: 0 results
4. TSUCHIYA Shigeru (30124605)

# of Collaborated Projects: 1 results

# of Collaborated Products: 5 results
5. SAKAMOTO Osamu (20333809)

# of Collaborated Projects: 1 results

# of Collaborated Products: 0 results
6. Hayashi Emiko (10620130)

# of Collaborated Projects: 1 results

# of Collaborated Products: 0 results
7. 佐野誠 (70339323)

# of Collaborated Projects: 1 results

# of Collaborated Products: 0 results
8. 西増弘志 (00467044)

# of Collaborated Projects: 1 results

# of Collaborated Products: 0 results
9. 加藤格 (10610454)

# of Collaborated Projects: 1 results

# of Collaborated Products: 0 results
10. 阿久津英憲 (50347225)

# of Collaborated Projects: 1 results

# of Collaborated Products: 0 results
11. 熊坂夏彦 (80525527)

# of Collaborated Projects: 1 results

# of Collaborated Products: 0 results
12. 横溝陵 (90773276)

# of Collaborated Projects: 1 results

# of Collaborated Products: 0 results
13. 和田美加子 (50810090)

# of Collaborated Projects: 1 results

# of Collaborated Products: 0 results
14. Minegishi Tomoko

# of Collaborated Projects: 1 results

# of Collaborated Products: 2 results
15. Akiba Yumi

# of Collaborated Projects: 1 results

# of Collaborated Products: 0 results
16. WADA Taizo

# of Collaborated Projects: 0 results

# of Collaborated Products: 1 results
17. IMAI Kohsuke

# of Collaborated Projects: 0 results

# of Collaborated Products: 1 results
18. KAWAI Toshinao

# of Collaborated Projects: 0 results

# of Collaborated Products: 2 results
19. ONUMA Masaei

# of Collaborated Projects: 0 results

# of Collaborated Products: 1 results
20. 宮戸真美

# of Collaborated Projects: 0 results

# of Collaborated Products: 1 results
21. 呉繁夫

# of Collaborated Projects: 0 results

# of Collaborated Products: 1 results
22. 真部淳

# of Collaborated Projects: 0 results

# of Collaborated Products: 1 results
23. 松本直通

# of Collaborated Projects: 0 results

# of Collaborated Products: 1 results
24. 滝田順子

# of Collaborated Projects: 0 results

# of Collaborated Products: 2 results
25. 柴田理恵

# of Collaborated Projects: 0 results

# of Collaborated Products: 1 results
26. 峯岸正好

# of Collaborated Projects: 0 results

# of Collaborated Products: 1 results
27. 山田雅文

# of Collaborated Projects: 0 results

# of Collaborated Products: 1 results
28. 辻本信一

# of Collaborated Projects: 0 results

# of Collaborated Products: 1 results

UCHIYAMA Toru 内山 徹

Research Projects

Research Products

Co-Researchers

特異的リガンドを使用した造血幹細胞へのin vivo遺伝子送達ベクター開発

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Development of a novel gene therapy strategy using base editing for hyper-IgE syndrome caused by STAT3 mutationsPrincipal Investigator

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Elucidation of 3D genome architecture of human placental and endometrial cells

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Development of a hematopoietic stem cell-targeted lentiviral vector system

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Investigation of molecular biology of CNS leukemia

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Genome editing-based gene therapy for primary immunodeficiency caused by dominant gain-of-function mutations.Principal Investigator

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Development of novel CAR-T cell therapy using a cancer-testis antigen CRT2

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Comprehensive analysis of somatic and germline genomic alterations for atypical leukemia in children

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Establishment of Baboon envelope pseudotyped LV vector system to maintain the stemness in stem cell gene therapy.Principal Investigator

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Foamy virus vector-mediated gene transfer into long-term HSC in the gene therapy for primary immunodeficiencies.Principal Investigator

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Homologous recombination-mediated gene correction in iPS cells o Ornithine transcarbamylase deficiency

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Homologous recombination-mediated gene correction for Adenosine deaminase deficiencyPrincipal Investigator

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[Book] 先端治療技術の実用化と開発戦略2017

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Total Pages

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UCHIYAMA Toru 内山徹

[Journal Article] 遺伝子治療開発研究ハンドブック　第1章 Ex vivo遺伝子治療, 第1節5. Genotoxicity.2023

[Journal Article] 原発性免疫不全症候群　診療の手引き　第1章複合免疫不全 B アデノシン・デアミナーゼ欠損症2023

[Journal Article] ゲノム編集の最新技術と医薬品・遺伝子治療・農業・水産物・有用物質生産への活用　第３章　遺伝子治療におけるゲノム編集技術の活用と課題対策　第5節　ゲノム編集技術による遺伝子治療の開発2023