国立研究開発法人 国立成育医療研究センター National Center for Child Health and Development

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Scholar & Enterprise

Scholar & Enterprise

Department of Human Genetics

We aim to identify causative genes for child intractable hereditary diseases and analyze their functions to develop new gene-based therapeutic treatments.

1. Stem cell gene therapy for primary immunodeficiencies

In collaboration with the National Institutes of Health (USA), we are conducting a clinical trial of stem cell gene therapy for chronic granulomatous disease using a retroviral vector. We also coordinate clinical trials of lentiviral-based gene therapy for Wiskott-Aldrich syndrome and X-linked severe combined immunodeficiency. We employ next-generation DNA sequencing and digital polymerase chain reactions (PCR) to evaluate the safety of gene therapy clinical trials, to ensure quality control of the clinical vectors and to analyze vector integration sites.

2. Establishment of disease-specific iPS cells

We establish specific iPS cells from peripheral blood or skin fibroblasts obtained from patients with intractable hereditary diseases such as primary immunodeficiencies and congenital metabolic disorders.
We perform detailed morbidity analysis and investigate inheritable genetic-modification using established iPS cells.

3. Gene therapy for congenital metabolic disorders

Lysosomal diseases including Niemann-Pick disease and mucopolysaccharidosis are good targets for gene therapy. We are currently developing a new strategy of Adeno-associated virus (AAV) vector-mediated gene delivery for these diseases.

4. Basic research of viral vectors

We carry out the cloning of transcriptional repressors of human origin and study the inhibition mechanism of gene expression using retroviral vectors.
We are developing safer viral vectors with high curative effects for future gene therapy clinical trials.

5. NCCHD BioBank

We store iPS cells from child intractable hereditary diseases as a bioresource in the NCCHD BioBank and support the BioBank in various research endeavors, including drug design and development.




  • Toru Yasuda
  • Akane Miura



  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.


  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


  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


  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.


  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.


  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.


  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


  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.


  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.


  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.