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成育遺伝研究部

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ごあいさつ

小野寺先生の顔写真遺伝子治療に関わって、早20年以上の月日が経ちます。 卒後5年目の時、地方の病院で小児医として勤務しながら、治療法のない病気の子ども達を見るたびに「何か根本的に病気を治せる方法はないだろうか」と思い悩み、「画期的な治療法」を求めて基礎の門を叩いた30歳の時です。それから20余年、今では遺伝子治療もかなりのコンセンサスが得られるようになりましたが、当時は、まだ、遺伝子治療があまり知られていない頃で、学会等で「どのような研究をなさっていますか?」の質問に「遺伝子治療」と答えるのは、たいそう恥ずかしいものがありました。ただ、当時より始めたレトロウイルスベクターの研究は今でもライフ・ワークとして続けており、「継続することの重要性」をこの年になって改めて感じております。
私は、これまで北海道大学でのアデノシン・デアミナーゼ(ADA)欠損症に対する遺伝子治療や筑波大での再発白血病に対するHSV-TK遺伝子導入ドナーリンパ球輸注療法を行ってきました。確かに、個人的には満足のいく部分もありますが、総合的にみるとやはり多くの問題点や改善点があり、その解決策を模索しておりました。そのような中、幸運にも平成21年にこの成育遺伝研究部部長の職を拝命致し、さらに、平成22年より当センター病院の免疫科の医長も併任することになりました。
私がここで求める研究スタイルは、単にことばだけのTranslational Researchではなく、真の意味で病院と研究所が一体となって進める臨床研究であり、当センターが中心となって小児難治性疾患に対する遺伝子治療を実施していくことで、我が国の包括的な遺伝子治療実施体制を構築できるものと信じております。
今後は、このホームページを通して当センターが計画している原発性免疫不全症に対する遺伝子治療臨床研究の進捗状況や国内外の遺伝子治療に関する興味あるニュースを配信していく予定ですので、皆様の遺伝子治療に関する忌憚のないご意見ならびにご質問等を頂ければと考えております。
また、私がそうであったように「新しいもの」を求める若い研究者は大歓迎です。まずは連絡ください。皆様方の問題意識を共有できればと考えております。
それでは、今後ともよろしくお願い致します。

成育遺伝研究部 部長　小野寺 雅史

E-mail：genecell@ncchd.go.jp


研究部紹介

成育遺伝研究部は、小児の難治性疾患に対する原因遺伝子の同定とその機能を解析することで疾患に対する新しい治療法を開発することを目的としています。特に、小児の原発性免疫不全症に対する診断法の開発と治療法の確立は、当センター病院と一体となって取り組んでいる主要な研究テーマです。
また、安全で効率の良いウイルスベクターの開発や各種幹細胞の遺伝子導入法の開発などの基礎研究も積極的に進めております。
遺伝子治療に興味のある方(研究希望の方)、幹細胞への遺伝子導入にお困りの方はお気軽にご連絡ください。


研究内容

原発性免疫不全症に関する研究

  1. 原発性免疫不全症の新規診断法の確立
  2. 原発性免疫不全症に対する造血幹細胞遺伝子治療の実施

幹細胞を用いた研究

  1. ヒト造血幹細胞のstemnessに関する研究
  2. ヒト造血幹細胞における遺伝子改変技術の改良

遺伝子治療臨床試験の安全性･有効性の検討

  1. 遺伝子導入細胞の生体内分布及び遺伝子発現解析
  2. 次世代シーケンサを用いたベクター導入部位解析

スタッフ

成育遺伝研究部

小野寺 雅史（部長）

疾患遺伝子構造研究室

内山 徹（室長）

部室員

安田 徹（研究員）
森下 真由（研究員）

規制および資料公開

遺伝子細胞治療に関する規制および学会等での資料を、「規制および資料公開」のページで公開します。本ページで提供する情報は、当研究部の許可無くコピー、転載・転用など二次的に利用することを禁じます。


FACS CORE LAB

成育遺伝研究部では、FACS COREを管理しています。
FACSをご希望の方はFACS CORE LABのページをご覧ください。

業績

2023

  1. 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.　Melting temperature mapping method in children: Rapid identification of pathogenic microbes. J Infect Chemother. 2023 doi: 10.1016/j.jiac.2023.11.024. Online ahead of print.
  2. 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. Congenital anaemia associated with loss-of-function variants in DNA polymerase epsilon 1. J Med genet. 2023 doi: 10.1136/jmg-2023-109444.
  3. 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. Myelodysplasia after clonal hematopoiesis with APOBEC3-mediated CYBB inactivation in retroviral gene therapy for X-CGD. Mol Ther. 2023 Sep 13:S1525-0016(23)00491-4. doi: 10.1016/j.ymthe.2023.09.004.
  4. Kaizuka A, Tokuda Y, Morooka S, Gocho Y, Funaki T, Uchiyama T, Hirai Y, Yasumi T, Maekawa T, Kubota M, Ishiguro A. Pediatric Hemophagocytic lymphohistiocytosis after concomitant administration of SARS-CoV-2 vaccine and influenza vaccine. J infect Chemother. 2023 Aug 30:S1341-321X(23)00201-5 doi; 10.1016/j.jiac.2023.08.05.
  5. Komeno Y, Uchiyama T, Kawano F, Kurihara Y, Kurokawa M, Ohara O, Kunishima S, Ishiguro A. Inherited macrothrombovytopenia due to a novel splice donor site mutation in ITGB3. Ann Hematol. 2023 102: 2947-2949. doi: 10.1007/s00277-023-05387-z
  6. Onodera M, Uchiyama T, Ariga T, Yamada M, Miyamura T, Arizono H, Morio T. Safety and efficacy of elapegademase in patients with adenosine deaminase deficiency: A multicenter, open-label, single-arm, phase 3, and postmarketing clinical study. Immun Inflamm Dis. 2023 Jul;11(7):e917. doi: 10.1002/iid3.917.
  7. Yasuda T, Uchiyama T, Watanabe N, Ito N, Nakabayashi K, Mochizuki H, Onodera M. Peripheral immune system modulates Purkinje cell degeneration in Niemann-Pick disease tyepe C1. Life Scie Alliance. 6: e202201881, 2023. doi: 10.26508/lsa.202201881.
  8. Kouno H, Kunishima S, Takebe J, Uchiyama T, Kaname T, Ishiguro A. Measurement of immature platelet fraction is useful in the differential diagnosis of MYH9 disorders. Int J Lab Hematol. 2023 doi: 10.1111/ijlh.14123.
  9. Shirai R, Osumi T, Keino D, Nakabayashi K, Uchiyama T, Sekiguchi M, Hiwatari M, Yoshida M, Yoshida K, Yamada Y, Tomizawa D, Takae S, Kiyokawa N, Matsumoto K, Yoshioka T, Hata K, Hori T, Suzuki N, Kato M. Minimal residual disease detection by mutation-specific droplet digital PCR for leukemia/lymphoma. Int J Hematol. 2023 Jun 117(6):910-918. doi:10.1007/s12185-023-03566-2.
  10. Tomomasa D, Isoda T, Mitsuiki N, Nishimura A, Uda K, Uchiyama T, Yamashita M, Kamiya T, Endo A, Takagi M, Imai K, Cawan MJ, Morio T, Kanegane H. Successful TCRab/CD19-depleted hematipoietic stem cell transplantation for a patient with artemis deficiency. J Pediatr Hematol Oncol. 2023 Mar 1;45(2):e285-e289 doi:10.1097/MPH.0000000000002522.
  11. Yamano S, Iguchi A, Ishikawa K, Sakamoto A, Uchiyama T, YAnagi K, Kaname T, Kunishima S, Ishiguro A. Splenectomy as an effective treatment for macrothrombocytopenia in Takenouchi-Kosaki syndrome. In J Hematol. 2023 Apr 117(4):622-625. doi:10.1007/s12185-022-03491-w.
  12. Mashima R, Ohira M, Okuyama T, Onodera M, Takada S. A novel mucopolysaccharidosis type II mouse model with an iduronate-2-sulfatase-P88L mutation. Sci Rep. 2023 May 15;13(1):7865. doi: 10.1038/s41598-023-34541-w.
  13. Irikura R, Nishizawa H, Nakajima K, Yamanaka M, Chen G, Tanaka K, Onodera M, Matsumoto M, Igarashi K. Ferroptosis model system by the re-expression of BACH1. J Biochem. 2023 Apr 24:mvad036. doi: 10.1093/jb/mvad036.

2022

  1. Matsukawa Y, Isshiki K, Osumi T, Fujiyama S, Fukushima H, Uchiyama T, Yamada M, Deguchi T, Imadome K, Matsumoto K, Tomizawa D, Takada H, Onodera M, Kato M. Successful hematopoietic stem cell transplantation with reduced dose of busulfan for Omenn synsdrome. Blood cell Therapy. 2022 May 5(3):75-78. doi:10.31547/bct-2021-021.
  2. Takeuchi I, Yanagi K, Takada S, Uchiyama T, Igarashi A, Matsuoka R, Yoshioka T, Saito H, Kawai T, Miyaji Y, Inuzuka Y, Matsubara Y, Ohya Y, Shimizu T, Matsumoto K, Arai K, Nomura I, Kaname T, Morita H. Identification of STAT6 gain-of-function variant that exacerbated multiple allergic symptoms. J. Allergy Clin Immunol. 2022 May 151(5):1402-1409.e6. doi:10.1016/j.jaci2022.12.802.
  3. Naiki Y, Miyado M, Shindo M, Horikawa R, Hasegawa Y, Katsumata N, Takada S, Akutsu H, Onodera M, Fukami M. Adeno-Associated Virus-Mediated Gene Therapy for Patients' Fibroblasts, Induced Pluripotent Stem Cells, and a Mouse Model of Congenital Adrenal Hyperplasia. Hum Gene Ther. 2022 Aug 33(15-16):801-809. doi:10.1089/hum.2022.005.
  4. Ochiai S, Hayakawa I, Ohashi E, Hmano S, Miyata Y, Sakuma H, Hogetsu K, Gocho Y, Ogura M, Uchiyama T, Abe Y. Fatal X-linked lymphoproliferative disease type 1-associated limbic encephalitis with positive anti-alpha-amino-3-hydroxy-methyl-4-isoxazolepropionic acid receptor antibody. Brain Dev. 2022 Oct 44(9):630-634. doi:10.1016/j.braindev.2022.06.004.

2021

  1. 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, Ariga T, Onodera M. Nonconditioned ADA-SCID gene therapy reveals ADA requirement in the hematopoietic system and clonal dominance of vector-marked clones. Molecular Therapy: Methods & Clinical Development 2021 Oct 16;23:424-433. doi: 10.1016/j.omtm.2021.10.003.
  2. Ishikawa T, Okai M, Mochizuki E, Uchiyama T, Onodera M, Kawai T. Bacillus Calmette-Guerin (BCG) infections at high frequency in both AR-CGD and X-CGD patients following BCG vaccination. Clin Infect Dis 2021 Nov 2;73(9):e2538-e2544. doi: 10.1093/cid/ciaa1049.
  3. Ishikawa K, Uchiyama T, Kaname T, Kawai T, Ishiguro A. Autoimmune hemolytic anemia associated with Takenouchi-Kosaki syndrome. Pediatr Int. 2021 Dec;63(12):1528-1530. doi: 10.1111/ped.14651.
  4. Kanamaru Y, Uchiyama T, Kaname T, Yanagi K, Ohara O, Kunishima S, Ishiguro A. ETV6-related thrombocytopenia associated with a transient decrease in von Willebrand factor. Int J Hematol. 2021. Aug;114(2):297-300. doi: 10.1007/s12185-021-03136-4.
  5. Ishikawa T, Tamura E, Kasahara M, Uchida H, Higuchi M, Kobayashi H, Shimizu H, Ogawa E, Yotani N, Irie R, Kosaki R, Kosaki K, Uchiyama T , Onodera M, Kawai T. Severe Liver Disorder Following Liver Transplantation in STING-Associated Vasculopathy with Onset in Infancy. J Clin Immunol. 2021 Jul;41(5):967-974. doi: 10.1007/s10875-021-00977-w.
  6. Uchiyama T, Kawakami S, Masuda H, Yoshida K, Niizeki H, Mochizuki E, Edasawa K, Ishiguro A, Onodera M. A Distinct Feature of T Cell Subpopulations in a Patient with CHARGE Syndrome and Omenn Syndrome. J Clin Immunol 2021 Jan;41(1):233-237. doi: 10.1007/s10875-020-00875-7.
  7. Ohira M, Kikuchi E, Mizuta S, Yoshida N, Onodera M, Nakanishi M, Okuyama T, Mashima R. Production of therapeutic iduronate-2-sulfatase enzyme with a novel single-stranded RNA virus vector. Genes Cells 2021 Nov;26(11):891-904. doi: 10.1111/gtc.12894.
  8. Kataura T, Tashiro E, Nishikawa S, Shibahara K, Muraoka Y, Miura M, Sakai S, Katoh N, Totsuka M, Onodera M, Shin-Ya K, Miyamoto K, Sasazawa Y, Hattori N, Saiki S, Imoto M. A chemical genomics-aggrephagy integrated method studying functional analysis of autophagy inducers. Autophagy 2021 Aug;17(8):1856-1872. doi: 10.1080/15548627.2020.1794590.
  9. Nishizawa H, Matsumoto M, Chen G, Ishii Y, Tada K, Onodera M, Kato H, Muto A, Tanaka K, Igarashi K. Lipid peroxidation and the subsequent cell death transmitting from ferroptotic cells to neighboring cells. Cell Death 2021 Mar 29;12(4):332. doi: 10.1038/s41419-021-03613-y.
  10. Azami Y, Tsuyama N, Abe Y, Sugai-Takahashi M, Kudo KI, Ota A, Sivasundaram K, Muramatsu M, Shigemura T, Sasatani M, Hashimoto Y, Saji S, Kamiya K, Hanamura I, Ikezoe T, Onodera M, Sakai A. Chromosomal translocation t(11;14) and p53 deletion induced by the CRISPR/Cas9 system in normal B cell-derived iPS cells. Sci Rep 2021 Mar 4;11(1):5216. doi: 10.1038/s41598-021-84628-5.
  11. Matsukawa Y, Isshiki K, Osumi T, Fujiyama S, Fukushima H, Uchiyama T, Yamada M, Deguchi T, Imadome KI, Matsumoto K, Tomizawa D, Takada H, Onodera M, Kato M. Successful hematopoietic stem cell transplantation with reduced dose of busulfan for Omenn syndrome. Blood Cell Ther [in press]

2020

  1. Takeuchi I, Kawai T, Nambu M, Migita O, Yoshimura S, Nishimura K, Yoshioka T, Ogura M, Kyodo R, Shimizu H, Ito S, Kato M, Onodera M, Hata K, Matsubara Y, Arai K. X-linked inhibitor of apoptosis protein deficiency complicated with Crohn's disease-like enterocolitis and Takayasu arteritis: A case report. Clin Immunol.2020 Aug;217:108495. doi: 10.1016/j.clim.2020.108495.
  2. Suzuki E, Kobori Y, Katsumi M, Ushijima K, Uchiyama T, Okada H, Miyado M, Fukami M. 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. Reprod Med Biol. 2020 Mar 2; 19(2):178-181. doi: 10.1002/rmb2.12321.
  3. Uchida H, Sakamoto S, Shimizu S, Takeda M, Yanagi Y, Fukuda A, Uchiyama T, Irie R, Kasahara M. Efficacy of Antithymocyte Globulin Treatment for Severe Centrilobular Injury Following Pediatric Liver Transplant: Clinical Significance of Monitoring Lymphocyte Subset. Exp Clin Transplant. 2020 Jun;18(3):325-333. doi: 10.6002/ect.2019.0387.
  4. 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 KI, Arai K, Deguchi T, Imai K, Yuza Y, Matsumoto K, Onodera M, Kanegane H, Tomizawa D, Kato M. A prospective study of allogeneic hematopoietic stem cell transplantation with post-transplantation cyclophosphamide and anti-thymoglobulin from HLA-mismatched related donors for non-malignant diseases. Biol Blood Marrow Transplant, 2020 Nov; 26(11): e286-e291. doi: 10.1016/j.bbmt.2020.08.008.
  5. Yamaguchi T, Uchida E, Okada T, Ozawa K, Onodera M, Kume A, Shimada T, Takahashi S, Tani K, Nasu Y, Mashimo T, Mizuguchi H, Mitani K, Maki K. Aspects of gene therapy products using current genome-editing technology in Japan. Hum Gene Ther. 2020 Oct; 31(19-20):1043-1053. doi: 10.1089/hum.2020.156.
  6. Ishikawa T, Okai M, Mochizuki E, Uchiyama T, Onodera M, Kawai T. BCG infections at high frequency in both AR-CGD and X-CGD patients following BCG vaccination. Clin Infect Dis. 2020 Jul 27; ciaa 1049. doi: 10.1093/cid/ciaa1049.
  7. Oka Y, Hamada M, Nakazawa Y, Muramatsu H, Okuno Y, Higasa K, Shimada M, Takeshima H, Hanada K, Hirano T, Kawakita T, Sakaguchi H, Ichimura T, Ozono S, Yuge K, Watanabe Y, Kotani Y, Yamane M, Kasugai Y, Tanaka M, Suganami T, Nakada S, Mitsutake N, Hara Y, Kato K, Mizuno S, Miyake N, Kawai Y, Tokunaga K, Nagasaki M, Kito S, Isoyama K, Onodera M, Kaneko H, Matsumoto N, Matsuda F, Matsuo K, Takahashi Y, Mashimo T, Kojima S, Ogi T. Digenic mutations in ALDH2 and ADH5 impair formaldehyde clearance and cause a multisystem disorder, AMeD syndrome. Sci Adv. 2020 Dec 18;6(51): eabd7197. doi: 10.1126/sciadv.abd7197.

2019

  1. Kimura Y, Shofuda T, Higuchi Y, Nagamori I, Oda M, Nakamori M, Onodera M, Kanematsu D, Yamamoto A, Katsuma A, Suemizu H, Nakano T, Kanemura Y, Mochizuki H. Human Genomic Safe Harbors and the Suicide Gene-Based Safeguard System for iPSC-Based Cell Therapy. Stem Cells Transl Med. 2019 Mar 19. doi: 10.1002/sctm.18-0039.
  2. Narumoto S, Sakamoto S, Sasaki K, Hirata Y, Fukuda A, Uchiyama T, Irie R, Yoshida T, Kasahara M. ABO-incompatible liver transplantation for children under 2 years of age: A case report and a single-center review. Pediatr Transplant. 2019 23:e13308. doi: 10.1111/petr.13308.
  3. Tsuchida M, Kirino Y, Soejima Y, Onodera M, Arai K, Tamura E, Ishikawa T, Kawai T, Uchiyama T, Nomura S, Kobayashi D, Taguri M, Mitsuhashi T, Takata A, Miyake N, Nakajima H, Miyake S, Matsumoto N. Haploinsufficiency of A20 caused by a novel nonsense variant or entire deletion of TNFAIP3 is clinically distinct from Behçet disease. Arthritis Res Ther. 21: 137, 2019 doi: 10.1186/s13075-019-1928-5
  4. Hosaka S, Kobayashi C, Saito H, Imai-Saito A, Suzuki R, Iwabuchi A, Kato Y, Jimbo T, Watanabe N, Onodera M, Imadome KI, Masumoto K, Nanmoku T, Fukushima T, Kosaki K, Sumazaki R, Takada H. Establishment of immunity against Epstein-Barr virus infection in a patient with CHARGE/complete DiGeorge syndrome after peripheral blood lymphocyte transfusion. Pediatr Transplant. 23:e13424. 2019 doi: 10.1111/petr.13424.
  5. Tomono T, Hirai Y, Chono H, Mineno J, Ishii A, Onodera M, Tamaoka A, Okada T. Infectivity Assessment of Recombinant Adeno-Associated Virus and Wild-Type Adeno-Associated Virus Exposed to Various Diluents and Environmental Conditions. Hum Gene Ther Methods. 30: 137-143, 2019.
  6. Kikuchi T, Nakae J, Kawano Y, Watanabe N, Onodera M, Itoh H. Foxo in T Cells Regulates Thermogenic Program through Ccr4/Ccl22. iScience. 22: 81-96, 2019.
  7. 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. Low prevalence of maternal microchimerism in peripheral blood of Japanese children with type I diabetes. Diabet Med. Doi: 1007/s12185-019-02795-8
  8. Shirai R, Osumi T, Terashima K, Kiyotani C, Uchiyama M, Tsujimoto S, Yoshida M, Yoshida K, Uchiyama T, Tmizawa D, Shioda Y, Sekiguchi M, Watanabe K, Keino D, Ueno-Yokohata H, Ohki K, Takita J, Ito S, Deguchi T, Kiyokawa N, Ogiwara H, Hishiki T, Ogawa S, Okita H, Matsumoto K, Yoshioka T, Kato M. High prevalence of SMARCB1 constitutional abnormalities including mosaicism in malignant rhabdoid tumours. Eur J Hum Genet 2020 Mar 26. doi: 10.1038/s41431-020-0614-z.
  9. Saito T, Ito Y, Samura O, Aoki H, Uchiyama T, Okamoto A, Hata K. Direct assessment of a single-cell DNA using crudely purified live cells: a proof of concept for noninvasive prenatal definitive diagnosis. J Mol Diagn. 22: 132-140, 2020. doi: 10.1016/j.jmoldx.2019.10.006.
  10. 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. Comparison of clonazepam and levetiracetam in children for prevention of busulfan-induced seizure in hematopoietic stem cell transplantation. Int J Hematol. 111: 463-466, 2020.
  11. 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; Japanese Study Group of Insulin Therapy for Childhood and Adolescent Diabetes. Low prevalence of maternal microchimerism in peripheral blood of Japanese children with type 1 diabetes. Diabet Med. 2019 Dec 23: doi: 10.1111/dme.14221.

2018

  1. Kamei K, Miyairi I, Ishikura K, Ogura M, Shoji K, Funaki T, Ito R, Arai K, Abe J, Kawai T, Onodera M, Ito S. Prospective Study of Live Attenuated Vaccines for Patients with Nephrotic Syndrome Receiving Immunosuppressive Agents. J Pediatr, S0022-3476 (17) 31756-0, 2018.
  2. Nishi K, Kawai T, Kubota M, Ishiguro A, Onodera M. X-linked agammaglobulinemia complicated with pulmonary aspergillosis. Pediatr Int 1: 90-92, 2018.
  3. Osumi T, Tsujimoto S, Nakabayashi K, Taniguchi M, Shirai R, Yoshida M, Uchiyama T, Nagasawa J, Goyama S, Yoshida T, Tomizawa T, Kurokawa M, Matsubara Y, Kiyokawa N, Hata K, Kato M. Somatic MECOM mosaicism in a patient with congenital bone marrow failure without a radial abnormality. Pediatr Blood Cancer. 65: e26959, 2018
  4. Tsujimoto S, Osumi T, Uchiyama M, Shirai R, Moriyama T, Nishii R, Yamada Y, Kudo K, Sekiguchi M, Arakawa Y, Yoshida M, Uchiyama T, KTerui, Ito S, Koh K, Takita J, Ito E, Tomizawa D, Manabe A, Kiyokawa N, Yang J, Kato M. Diplotype analysis of NUDT15 variants and 6-mercaptopurine sensitivity in pediatric lymphoid neoplasms. Leukemia. 32: 2710-2714, 2018.
  5. Okano T, Imai K, Uchiyama T, Morio T. Hematopoietic stem cell transplantation for progressive combined immunodeficiency and lymphoproliferation in Activated PI3Kd syndrome type 1. J Allergy Clin Immunol. 2018 (in press)
  6. Osumi T, Tsujimoto S, Tamura M, Uchiyama M, Nakabayashi K, Okamura K, Toshida M, Tomizawa D, Watanabe A, Takahashi H, Hori T, Yamamoto S, Hamamoto K, Migita M, Ogata-Kawata H, Uchiyama T, Kizawa H, Ueno-Yokohata H, Shirai R, Seki M, Ohki K, Inukai T, Ogawa S, Kitamura T, Matsumoto K, Hata K, Kiyokawa N, Goyama S, Kato M. Recurrent RARB translocation in Acute Promyelocytic leukemia lacking RARA translocation. Cancer Res. 78: 4452-4458, 2018
  7. Osumi T, Tomizawa D, Kawai T, Sako M, Inoue E, Takimoto T, Tamura E, Uchiyama T, Imadome KI, 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. A prospective study of allogeneic transplantation from unrelated donors for chronic granulomatous disease with target busulfan-based reduced-intensity conditioning. Bone Marrow Transplant. 018 Jun 29. doi: 10.1038/s41409-018-0271-9
  8. Shoji K, Kawai T, Onodera M, Tsutsumi Y, Nosaka S, Miyairi I. Multiple osteolytic lesions on the skull of a girl with Mendelian susceptibility to mycobacterial disease. Pediatr Int. 60: 1043-1044, 2018.
  9. Tomono T, Hirai Y, Okada H, Miyagawa Y, Adachi K, Sakamoto S, Kawano Y, Chono H, Mineno J, Ishii A, Shimada T, Onodera M, Tamaoka A, Okada T. Highly Efficient Ultracentrifugation-free Chromatographic Purification of Recombinant AAV Serotype 9. Mol Ther Methods Clin Dev. 11: 180-190, 2018. 32:2710-2714, 2018
  10. Tsujimoto S, Osumi T, Uchiyama M, Shirai R, Moriyama T, Nishii R, Yamada Y, Kudo Km Sekiguchi M, Arakawa Y, Yoshida M, Uchiyama T, Terui K, Ito S, Koh K, Takita J, Tomizawa D, Manabe A, Kiyokawa N, N. Yang JJ, Kato M. Diplotype analysis of NUD15 variants and 6-mercaptopurine sensitivity in pediatric lymphoid neoplasms. Leukemia, 32:2710-2714, 2018
  11. Narumoto S, Sakamoto S, Sasaki K, Hirata Y, Fukuda A, Uchiyama T, Irie R, Yoshioka T, Kasahara M. ABO-incompatible liver transplantation for children under 2 years of age: A case report and a single center review. Pediatr. Transplant. Doi: 10.1111/petr.13308. 2018
  12. 小野寺雅史　原発性免疫不全症の遺伝子治療　医薬ジャーナル 54:59-66，2018.
  13. 小野寺雅史　遺伝子治療の新局面 - はじめに- 医学のあゆみ265: 323，2018.
  14. 内山徹　レトロウイルス、レンチウイルスベクター：特徴と作製法、最近の話題　医学のあゆみ265: 337，2018.
  15. 小野寺雅史　毛細血管拡張性運動失調症 産科と婦人科 85:649-653，2018.
  16. 小野寺雅史　ヘモグロビン異常症に対する造血幹細胞遺伝子治療 - βサラセミナ、鎌状赤血球症 - 血液フロンティア 28:768-774，2018.

2017

  1. Okuno M, Kasahara Y, Onodera M, Takubo N, Okajima M, Suga S, Watanabe N, Suzuki J, Ayabe T, Urakami T, Kawamura T, Kikuchi N, Yokota I, Kikuchi T, Amemiya S, Nakabayashi K, Hayashi K, Hata K, Matsubara Y, Ogata T, *Fukami M, Sugihara S. Nucleotide substitutions in CD101, the human homolog of a diabetes susceptibility gene in non-obese diabetic mouse, in patients with type 1 diabetes. Journal of Diabetes Investigation 8: 286-294, 2017.
  2. Osumi T, Kato M. Ouchi-Uchiyama M, Tomizawa D, Kataoka K, Fuji Y, Seki M, TAkita J, Ogawa S, Uchiyama T, Ohki K, Kiyokawa N. Blastic transformation of juvenile myelomonocytic leukemia caused by the copy number gain of onocogenic KRAS. Pediatr Blood Cancer 2017 doi: 10.1002/pbc.26496
  3. Goto F, Uchiyama T, Nakazawa Y, Kawai T, Imai K, Onodera M. Persistent impairment of T cell regeneration in a patient with activated PI3K d syndrome. J Clin Immunol. 2017 doi: 10.1007/s10875-017-0393-7

2016

  1. Ichida Y, Utsunomiya Y, Onodera M: Effect of the linkers between the zinc fingers in zinc finger protein 809 on gene silencing and nuclear localization. Biochemical and Biophysical Research Communications. 471: 533-538, 2016.
  2. Obayashi N, Arai K, Nakano N, Mizukami T, Kawai T, Yamamoto S, Shimizu H, Nunoi H, Shimizu T, Tang J, Onodera M: Leopard skin-like colonic mucosa: A novel endoscopic finding of chronic granulomatous disease-associated colits.　Journal of Pediatric Gastroenterology and Nutrition. 62: 56-59, 2016.
  3. Ichida Y, Utsunomiya Y, Onodera M: The third to fifth zinc fingers play an essential role in the binding of ZFP809 to the MLV-derived PBS. Biochemical and Biophysical Research Communications. 469: 490-494, 2016.
  4. Choong CJ, Sasaki T, Hayakawa H, Yasuda T, Baba K, Hirata Y, Uesato S, Mochizuki H. A　novel histone deacetylase 1 and 2 isoform-specific inhibitor alleviates　experimental Parkinson's disease. Neurobiol Aging. 37:103-16 2016.
  5. Tomono T, Hirai Y, Okada H, Adachi K, Ishii A, Shimada T, Onodera M, Tamaoka A, *Okada T: Ultracentrifugation-free chromatography-mediated large-scale purification of recombinant adeno-associated virus serotype 1 (rAAV1). Molecular Therapy Methods and clinical Development. 3: 15058, 2016.
  6. Nagaya M, Watanabe M, Kobayashi M, Nakano K, Arai Y, Asano Y, Takeishi T, Umeki I, Fukuda T, Yashima S, Takayanagi S, Watanabe N, Onodera M, Matsunari H, Umeyama K, *Nagashima H. A transgenic-cloned pig model expressing non-fluorescent modified Plum. Journal of Reproduction and Development. 62; 511-520, 2016
  7. Kawano Y, *Nakae J, Watanabe N, Kikuchi T, Tateya S, Tamori Y, Kaneko M, Abe T, Onodera M, Itoh H. Colonic Pro-Inflammatory Macrophages Cause Insulin Resistance in an Intestinal Ccl2/Ccr2-Dependent Manner. Cell Metabolism. 24: 295-310, 2016.
  8. Hasegawa　K, Yasuda T, Shiraishi C, Fujiwara K, Przedborski S, Mochizuki H, Yoshikawa K. Promotion of mitochondrial biogenesis by necdin protects neurons against mitochondrial insults. Nat Commun. 14;7:10943, 2016.
  9. Ikawa Y, Uchiyama T, Jagadeesh GJ, *Candotti F. 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. Gene therapy. 23:815-818. 2016.
  10. Naiki Y, Miyado M, Horikawa R, Katsumata N, Onodera M, Pang S, Ogata T, *Fukami M. Extra-adrenal induction of Cyp21a1 ameliorates systemic steroid metabolism in a mouse model of congenital adrenal hyperplasia. Endocrine Journal. 63: 897-904, 2016.

2015

  1. 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: Effects of enzyme replacement therapy on immune function in ADA deficiency patient. Clin Immunol 161: 391-393, 2015.
  2. Ichida Y, Utsunomiya Y, Yasuda T, Nakabayashi K, Sato T, Onodera M: Functional domains of ZFP809 essential for nuclear localization and gene silencing. PLOS ONE 10: e0139274, 2015.
  3. Ichida Y, Utsunomiya Y, Tomikawa J, Nakabayashi K, Sato T, Onodera M: Long time-course monitoring of ZFP809-mediated gene silencing in transgene expression driven by promoters containing MLV-derived PBS. Biosci Biotechnol Biochem 7:1-7, 2015.
  4. Kawai T, Arai K, Harayama S, Nakazawa Y, Goto F, Maekawa T, Tamura E, Uchiyama T, Onodera M: Severe and Rapid Progression in Very Early-Onset Chronic Granulomatous Disease-Associated Colitis. J Clin Immunol 35: 583-588, 2015.
  5. Yoshida W, Tomikawa J, Inaki M, Kimura H, Onodera M, Hata K, Nakabayashi K: An insulator element located at the cyclin B1 interacting protein 1 gene locus is highly conserved among mammalian species. PLOS ONE 10: e0131204, 2015.
  6. Takeuchi Y, Takeuchi E, Ishida T, Onodera M, Nakauchi H, Otsu M: Curative haploidentical BMT in a murine model of X-linked chronic granulomatous disease. Int J Hematol 102: 111-120, 2015.
  7. Watanabe M, Kobayashi1 M, Nagaya M, Matsunari H, Nakano K, Maehara M, Hayashida G, Takayanagi S, Sakai R, Umeyama K, Watanabe N, Onodera M, Nagashima H: Production of transgenic cloned pigs expressing the far-red fluorescent protein monomeric Plum. J. Reprod Dev 61: 169-177, 2015.
  8. Otsu M, Yamada M, Nakajima S, Kida M, Maeyama Y, Hatano N, Toita N, Takezaki S, Okura Y, Kobayashi R, Matsumoto Y, Tatsuzawa , Tsuchida F, Kato S, Kitagawa M, Mineno J, Hershfield MS, Bali P, Candotti F, Onodera M, Kawamura N, Sakiyama Y, Ariga T: Treatment Outcomes in Two Japanese Adenosine Deaminase-Deficiency Patients Treated by Stem Cell Gene Therapy with No Cytoreductive Conditioning. J Clin Immunol 35: 384-398, 2015.

2014

  1. Takeda K, Kawai T, Nakazawa Y, Komuro H, Yamamoto M, Shoji K, Morita K, Miyairi I, Katsuta T, Ohya Y, Ishiguro A, Onodera M: Augmentation of anti-tubercular therapy with interferon g in a patient with dominant partial interferon g. Clin. Immunol. 151: 25-28, 2014.
  2. Onuki K, Sugiyama H, Ishige K, Kawamoto T, Ota T, Ariizumi S, Yamato M, Kadota S, Takeuchi K, Ishikawa A, Onodera M, Onizawa K, Yamamoto M, Miyoshi E, Shoda J: Expression of N-acetylglucosaminyltransferase V in the subserosal layer correlates with postsurgical survival of pathological tumor stage 2 carcinoma of the gallbladder. J Gastroenterol 49:702-714, 2014.
  3. Akagi K, Kawai T, Watanabe N, Yokoyama M, Arai K, Harayama S, Oana S, Onodera M: A case of macrophage activation syndrome developing in a patient with chronic granulomatous disease-associated colitis. J Pediatr Hematol Oncol 36: e169-172, 2014.
  4. Lai CY, Yamazaki S, Okabe M, Suzuki S, Maeyama Y, Iimura Y, Onodera M, Kakuta S, Iwakura Y, Nojima M, Otsu M, Nakauchi H: Stage-specific roles for Cxcr4 signaling in murine hematopoietic stem/progenitor cells in the process of bone marrow repopulation. Stem Cells 32: 1929-1942, 2014.
  5. Looi CY, Sasahara Y, Watanabe Y, Satoh M, Hakozaki I, Uchiyama M, Wong WF, Du W, Uchiyama T, Kumaki S, Tsuchiya S, KureS. The open conformation of WASP regulates its nuclear localization and transcription in myeloid cells. Int Immunol. 26: 341-352, 2014
  6. Katsuya Y, Hojo M, Kawai S, Kawai T, Onodera M, Sugiyama H, Chronic granulomatous disease with pulmonary mass-like opacities secondary to hypersensitivity pneumonitis. J. Med. Case Rep.2014 Jul 2;8(1):242.　
  7. Shigeta T, Sakamoto S, Uchida H, Sasaki K, Hamano K, Kanazawa H, Fukuda A, Kawai T, Onodera M, Nakazawa A, Kasahara M: Basiliximab treatment for steroid-resistant rejection in pediatric patients with acute liver failure after liver transplantation. Pediatr. Transplant. 18: 860-867, 2014.
  8. Kawai T, Watanabe N, Yokoyama M, Nakazawa Y, Goto F, Uchiyama T, Higuchi M, Maekawa T, Tamura E, Nagasaka S, Hojo M, Onodera M.: Interstitial Lung Disease with Multiple Microgranulomas in Chronic Granulomatous Disease. J. Clin Immunol. 34: 933-940, 2014.
  9. Yamazaki Y, Yamada M, Kawai T, Morio T, Onodera M, Ueki M, Watanabe N, Takada H, Takezaki S, Chida N, Kobayashi I, Ariga T: Two Novel Gain-of-Function Mutations of STAT1 Responsible for Chronic Mucocutaneous Candidiasis Disease: Impaired Production of IL-17A and IL-22, and the Presence of anti-IL-17F Autoantibody. J. Immunol. 193: 48800-48807, 2014.
  10. Mori M, Onodera M, Morimoto A, Kosaka Y, Morio T, Notario GF Sharma S, Saji T: Palivizumab use in Japanese infants and children with immunocompromised conditions. Pediatr. Infect. Dis. J. 33: 1183-1185, 2014.

2013

  1. Kawai T, Watanabe N, Yokoyama M, Arai K, Oana S, Harayama S, Yasui K, Oh-ishi T, Onodera M: Thalidomide Attenuates Excessive Inflammation without Interrupting Lipopolysaccharide-driven Inflammatory Cytokine Production in Chronic Granulomatous Disease. Clin. Imunol. 2013;147:122-128
  2. Hamanaka S, Ooehara Jun, Morita Y, Ema H, Takahashi S, Miyawaki A, Otsu M, Yamaguchi T, Onodera M, Nakauchi H: Generation of transgenic mouse line expressing Kusabira Orange throughout body, including erythrocytes, by random segregation of provirus method. BBRC 2013;435:586-591
  3. Watanabe Y, Sasahara Y, Saoh 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. A case series of CAEBV of children and young adults treated with reduced-intensity conditioning and allogeneic bone marrow transplantation: a single center study. Eur J Haematol. 2013;91:242-248
  4. Tornack J, Seiler K, Grützkau A, Grün JR, Onodera M, Melchers F, Tsuneto M: Ectopic runx1 expression rescues tal-1-deficiency in the generation of primitive and definitive hematopoiesis. PLoS One 2013;8(7):e70116
  5. Yamamoto R, Morita Y, Ooehara J, Hamanaka S, Onodera M, Rudolph KL, Ema H, Nakauchi H:　Clonal analysis unveils self-renewing lineage-restricted progenitors generated directly from hematopoietic stem cells 2013;154:1112-1126
  6. Kobayashi M,Watanabe M, Matsunari H, Nakano K, Kanai T, Hayashida G, Matsumura Y, Kuramoto M, Sakai R, Arai Y, Umeyama K, Watanabe N, Onodera M, Nagaya M, Nagashima H:　20 generation and characterization of transgenic-cloned pigs expressing the far-red fluorescent protein monomeric plum.　Reprod Fertil 2013;26:124-125
  7. 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,Ishii:　NGene therapy model of X-linked severe combined immunodeficiency using a modified foamy virus vector. PLos One 2013;8:e71594

2012

  1. Kawano Y, Nakae J, Watanabe N, Fujisaka S, Iskandar K, Sekioka R, Hayashi Y, Tobe K, Kasuga M, Noda T, Yoshimura A, Onodera M, Itoh H: Loss of PDK1-Foxo1 signaling in myeloid cells predisposes to adipose tissue inflammation and insulin resistance. Diabetes 61: 1935-1948, 2012.
  2. Imagawa T, Takei S, Umebayashi H, Yamaguchi K, Itoh Y, Kawai T, Iwata N, Murata T, Okafuji I, Miyoshi M, Onoe Y, Kawano Y, Kinjo N, Mori M, Mozaffarian N, Kupper H, Santra S, Patel G, Kawai S, and Yokota S: Efficacy, Pharmacokinetics, and Safety of Adalimumab in Pediatric Patients with Juvenile Idiopathic Arthritis in Japan. Clin Rheumatol 31: 1713-1721, 2012.
  3. Kawai T, Nishikomori R, Izawa K, Murata Y, Tanaka N, Sakai, H Saito M, Yasumi T, Takaoka Y, Nakahata T, Mizukami T, Nunoi H, Kiyohara Y, Yoden A, Murata T, Sasaki, S Ito E, Akutagawa H, Kawai T, Imai C, Okada, S Kobayashi M, Heike T: Frequent somatic mosaicism of NEMO in T cells of patients with X-linked anhidrotic ectodermal dysplasia with immunodeficiency. Blood 119: 5458-5466, 2012．

2011

  1. Kawahara M, Chen J, Sogo T, Teng J, Otsu M, Onodera M, Nakauchi H, Ueda H, Nagamune T:Growth promotion of genetically modified hematopoietic progenitors using antibody/ c-Mpl chimera. Cytokine 55: 402-408, 2011.
  2. Maeyama Y, Otsu M, Kubo S, Yamano T, Iimura Y, Onodera M, Kondo S, Sakiyama Y, Ariga T: Intracellular estrogen receptor-binding fragment associated antigen 9 exerts in vivo tumor promoting effects via its coiled-coil region. Int J Oncology 39: 41-49, 2011.
  3. Fujisawa Y, Nabekura T, Kawachi Y, Otsuka F, Onodera M: Enforced ROR(gamma)t expression in haematopoietic stem cells increases regulatory T cell number, which reduces immunoreactivity and attenuates hypersensitivity in vivo. Asian Pac J Allergy Immunol 29: 86-93, 2011.
  4. Kunishima S, Kashiwagi H, Otsu M, Takayam N, Eto K, Onodera M, Miyajima Y, Takamatsu Y, Suzumiya J, Matsubara K, Tomiyama Y, S Hidehiko: Heterozygous ITGA2B R995W mutation inducing a constitutive activation of the aIIbb3 receptor affects proplatelet formation and causes congenital macrothrombocytopenia. Blood117: 5479-5484, 2011.
  5. Sugiyama H, Onuki K, Ishige K, Baba N, Ueda T, Matsuda S, Takeuchi K, Onodera M, Nakanuma Y, Yamato M, Yamamoto M, Hyodo I, Shoda J: Potent In Vitro and In Vivo Antitumor Activity of Sorafenib Against Human Intrahepatic Cholangiocarcinoma Cells. J Gastroenterol 46: 779-789, 2011.
  6. Kawai T, Kusakabe H, Seki A, Kobayashi S, Onodera M: Osteomyelitis due to triethoprim/sulfamethoxazole-resistant Edwardsiella tarda infection in a patient with X-linked chronic granulomatous disease. Infection 39: 171-173, 2011.

2010

  1. Tozuka Y, Kumon M, Wada E, Onodera M, Mochizuki H, Wada K: Material obesity impairs hippocampal BDNF production and spatial learning performance in young mouse offspring. Neurochem Int 57: 235-247, 2010.
  2. Hirata Y Hamanaka S, Onodera M: Transactivation of the dopamine receptor 3 gene by a single provirus integration results in development of B cell lymphoma in transgenic mice generated from retrovirally transduced embryonic stem cells. Blood115: 3930-3938, 2010.
  3. Miyamoto N, Tanaka R, Shimura H, Watanabe T, Mori H, Onodera M, Mochizuki H, Hattori N, Urabe T: Phosphodiesterase III inhibition promotes differentiation and survival of oligodendrocyte progenitors and enhances regeneration of ischemic white matter lesions in the adult mammalian brain. J Cereb Blood Flow Metab 30: 299-310, 2010.
  4. Kobayashi H, Shimada Y, Ikegami M, Kawai T, Sakurai K, Urashima T, Ijima M, Fujiwara M, Kaneshiro K, Ohashi T, Eto Y, Ishigaki K, Osawa M, Kyosen SO, Ida H: Prognostic factors for the late onset Pompe disease with enzyme replacement therapy: From our experience of 4 cases including an autopsy case. Mol Genet Metab100: 14-19, 2010.

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