Evaluation system for regenerative medicine of genetic disorders by using cloned pigs established from endoderm somatic stem cells

• Project/Area Number: 22390209
• Research Category: Grant-in-Aid for Scientific Research (B)
• Allocation Type: Single-year Grants
• Section: 一般
• Research Field: Pediatrics
• Research Institution: Kumamoto University
• Principal Investigator: ENDO Fumio 熊本大学, 大学院・生命科学研究部, 教授 (00176801)
• Co-Investigator(Kenkyū-buntansha): NAKAMURA Kimitoshi 熊本大学, 医学部附属病院, 講師 (30336234) ; MATSUMOTO Shiro 熊本大学, 医学部附属病院, 助教 (70467992)
• Project Period (FY): 2010 – 2012
• Project Status: Completed (Fiscal Year 2012)
• Budget Amount: ¥18,850,000 (Direct Cost: ¥14,500,000、Indirect Cost: ¥4,350,000); Fiscal Year 2012: ¥3,510,000 (Direct Cost: ¥2,700,000、Indirect Cost: ¥810,000); Fiscal Year 2011: ¥6,240,000 (Direct Cost: ¥4,800,000、Indirect Cost: ¥1,440,000); Fiscal Year 2010: ¥9,100,000 (Direct Cost: ¥7,000,000、Indirect Cost: ¥2,100,000)
• Keywords: 再生医学 / 移植・再生医療 / 糖尿病 / 発生・分化 / バイオテクノロジー / 幹分離法 / 内胚葉幹細胞 / 唾液腺 / 膵臓 / 肝臓

Research Abstract

We first isolated tissue stem cells originated from porcinesalivary gland. This clone was used for the generation of cloned pigs. Using this clonedpigs, we established a model system of cell-transplantation for the treatment of liverand pancreatic disorders. We also established a new culture system using variousconcentrations of amino acids in the medium.

Research Products

• [Journal Article] Calreticulin induces dilatedcardiomyopathy (2013)
  • Author(s): Lee D, Oka T, Hunter B, Robinson A, PappS, Nakamura K, Srisakuldee W, Nickel BE,Light PE, Dyck JRB, Lopaschuk GD,Kardami E, Opas M, and Michalak M
  • Journal Title: Plos One Volume: (inpress)
  • Peer Reviewed
• [Journal Article] VLCAD deficiency in a patient whorecovered from VF (2013)
  • Author(s): Yamamoto A, Nakamura K, Matsumoto S,Iwai M, Shigematsu Y, Tajima G, TsumuraM, Okada S, Mitsubuchi H, Endo F
  • Journal Title: but died suddenly ofan RSV infection Volume: (inpress)
  • Peer Reviewed
• [Journal Article] p.E66Q Mutation in the GLAGene is Associated with a High Risk ofCerebral Small-Vessel Occlusion inElderly Japanese Males (2013)
  • Author(s): Nakamura K, Sekijima Y, NakamuraK,Hattori K, Nagamatsu K, Shimizu Y,Yazaki M, Sakurai A, Endo F, FukushimaY, Ikeda S
  • Journal Title: Eur J Neurol Volume: (inpress)
  • Peer Reviewed
• [Journal Article] Newborn screening for Fabry disease in Japan:Prevalence and genotypes of Fabrydisease in a pilot study (2013)
  • Author(s): Inoue T, Hattori K, Ihara K, Ishii A,Nakamura K, Hirose S
  • Journal Title: J. Hum. Genet Volume: (inpress)
  • Peer Reviewed
• [Journal Article] Re-evaluation of the indications forliver transplantation in Wilson'sdisease based on the outcomes ofpatients referred to a transplantcenter (2013)
  • Author(s): Ohya Y, Okajima H, Honda M, HayashidaS, Suda H, Matsumoto S, Lee KJ,Yamamoto H, Takeichi T, Mitsubuchi H,Asonuma K, Endo F, Inomata Y
  • Journal Title: Pediatr Transplant Volume: 17(4) Pages: 369-737
  • Peer Reviewed
• [Journal Article] Current status of hepaticglycogen storage disease in Japan:clinical manifestations, treatmentsand long-term outcomes (2013)
  • Author(s): Kido J, Nakamura K, Matsumoto S,Mitsubuchi H, Ohura T, Shigematsu Y,Yorifuji T, Kasahara M, Horikawa R,Endo F
  • Journal Title: J Hum Genet Volume: 58(5) Pages: 285-92
  • Peer Reviewed
• [Journal Article] Current status of hepatic glycogen storage disease in Japan: clinical manifestations, treatments and long-term outcomes. (2013)
  • Author(s): Kido J
  • Journal Title: J. Hum. Genet. Volume: 未定
  • Peer Reviewed
• [Journal Article] Calreticulin induces dilated cardiomyopathy. (2013)
  • Author(s): Lee D
  • Journal Title: Plos One Volume: 未定
  • Peer Reviewed
• [Journal Article] VLCAD deficiency in a patient who recovered from VF, but died suddenly (2013)
  • Author(s): Yamamoto A
  • Journal Title: RSV infection Volume: 未定
  • Peer Reviewed
• [Journal Article] Identification of a novel mutation andprevalence study for fabry disease inJapanese dialysis patients (2012)
  • Author(s): Nishino T, Obata Y, Furusu A, HiroseM, Shinzato K, Hattori K, Nakamura K,Matsumoto T, Endo F, Kohno S
  • Journal Title: RenFail Volume: 34 Pages: 566-570
  • Peer Reviewed
• [Journal Article] Ohta T Effectof body mass index-z score on adverselevels of cardiovascular disease riskfactors (2012)
  • Author(s): Katsuren K, Nakamura K
  • Journal Title: Pediatr Int Volume: 54 Pages: 200-204
  • Peer Reviewed
• [Journal Article] Effect of body mass index-z score on adverse levels of cardiovascular disease risk factors. (2012)
  • Author(s): Katsuren K
  • Journal Title: Pediatr Int. Volume: 54 Issue: 2 Pages: 200-204
  • DOI: 10.1111/j.1442-200x.2011.03499.x
  • Peer Reviewed
• [Journal Article] Time-dependent changes in the plasmaamino acid concentration in diabetesmellitus (2011)
  • Author(s): Mochida T, Tanaka T, Shiraki Y, TajiriH, Matsumoto S, Shimbo K, Ando T,Nakamura K, Okamoto M, Endo F
  • Journal Title: Mol Genet Metab Volume: 103(4) Pages: 406-9
  • Peer Reviewed
• [Journal Article] Long-term outcome and intervention of urea cycle disorders in Japan (2011)
  • Author(s): Kido J, Nakamura K, Mitsubuchi H, Ohura T, Takayanagi M, Matsuo M, Yoshino M, Shigematsu Y, Yorifuji T, Kasahara M, Horikawa R, Endo F
  • Journal Title: J Inherit Metab Dis Volume: (Epub ahead of print)
  • Peer Reviewed
• [Journal Article] Newborn Screening for lysosomal disorders. (2011)
  • Author(s): Nakamura K, Hattori K, Endo F
  • Journal Title: Am J Med Genet. Volume: 157 Pages: 63-71
  • Peer Reviewed
• [Journal Article] Time-dependent changes in the plasma amino acid concentration in 3 diabetes mellitus. (2011)
  • Author(s): Mochida t, Endo F., et al.
  • Journal Title: Mol.Genet.Metab. Volume: (In press)
  • Peer Reviewed
• [Journal Article] Cerebral hemorrhage in Fabry's disease (2010)
  • Author(s): Nakamura K, Endo F., et al.
  • Journal Title: J Hum Genet. Volume: 55 Pages: 259-261
  • NAID: 10030734716
  • Peer Reviewed
• [Presentation] Screening for Fabry Disease in Japan (2012)
  • Author(s): Kimitoshi Nakamura, Fumio Endo
  • Organizer: 2012 Joint Conference of MedicalGenetics, Genomics & Korean LSDSymposium
  • Place of Presentation: Seoul, Korea
  • Year and Date: 2012-11-23
• [Presentation] 熊本市小児生活習慣病予防検診の現状～小児生活習慣病専門医の立場から (2012)
  • Author(s): 中村公俊
  • Organizer: 第43回全国学校保健 ・ 学校医大会シンポジウム 子どもたちの生活習慣病予防
  • Place of Presentation: ホテル日航熊本、熊本市
  • Year and Date: 2012-11-10
• [Presentation] Overview of Japan Newborn ScreeningExperience for Pompe Disease (2012)
  • Author(s): Kimitoshi Nakamura, Fumio Endo
  • Organizer: 14th AsiaLSD Symposium
  • Place of Presentation: BeijingChina
  • Year and Date: 2012-10-20
• [Presentation] Screening for Fabry Disease in Japan (2012)
  • Author(s): Kimitoshi Nakmaura, Fumio Endo
  • Organizer: 4th International Forum for LysosomalStorage Disease
  • Place of Presentation: TokyoPrince Hotel
  • Year and Date: 2012-10-05
• [Presentation] A patient ofGaucher's disease type 2 withrecurrent ARDS (2012)
  • Author(s): Shirou Matsumoto, Kiyoko Hattori,Kimitoshi Nakamura, HiroshiMitsubuchi1, Fumio Endo
  • Organizer: The 4th InternationalForum for Lysosomal Storage Disease &17th Japanese Society for. LysosomalDisease
  • Place of Presentation: Tokyo Prince Hotel
  • Year and Date: 2012-09-05
• [Presentation] Screening for Lysosomal StorageDisorders (2012)
  • Author(s): Kimitoshi Nakamura,Fumio Endo
  • Organizer: The 18 th Annual Meeting ofJapan Society of Gene Therapy
  • Place of Presentation: Hotel Kumamoto Terrsa
  • Year and Date: 2012-06-30
• [Presentation] Clinical experience and researches on metabolic disease (2012)
  • Author(s): 松本志郎、遠藤文夫
  • Organizer: ドイツハイデルベルグ小児病院リサーチカンファ
  • Place of Presentation: ハイデルベルグ大学、ドイツ
  • Year and Date: 2012-01-30
• [Presentation] Newborn Screening in Japan (2012)
  • Author(s): Kimitoshi Nakmaura, Fumio Endo
  • Organizer: The 10thAsia-Pacific Conference on HumanGenetics
  • Place of Presentation: Kuala Lumpur,Malaysia
• [Presentation] 生活習慣病患児における血中アミノ酸プロファイルおよび糖尿病発症予測の試み (2012)
  • Author(s): 松本志郎
  • Organizer: 第46回日本小児内分泌学会学術集会
  • Place of Presentation: 大阪国際会議場
• [Presentation] Treatment plans and genetic counseling for late-onset ornithine transcarbamylase deficiency. (2012)
  • Author(s): Daisuke Fujisawa
  • Organizer: 第54回日本先天代謝異常学会
  • Place of Presentation: 岐阜 じゅうろくプラザ
• [Presentation] 当院における急性肝不全23例の病因および予後の検討 (2011)
  • Author(s): 松本志郎、遠藤文夫
  • Organizer: 日本先天代謝異常学会・アジア先天代謝異常症シンポジウム
  • Place of Presentation: ホテルニューオータニ幕張、千葉市
  • Year and Date: 2011-11-24
• [Presentation] ISOLATION NESTIN-POSITIVE CELLS FROM NEONEATL PATIENT WITH HYPERINSULINEMIC HYPOGLYCEMIA OF INFANCY (2011)
  • Author(s): 松本志郎、遠藤文夫
  • Organizer: 第17回日本遺伝子治療学会
  • Place of Presentation: 九州大学セントラルホール、福岡
  • Year and Date: 2011-07-16
• [Presentation] Cell transplantation study for hereditary tyrosinemia type I model mice with mouse salivary gland derived progenitors (2011)
  • Author(s): 中村公俊、松本志郎、遠藤文夫
  • Organizer: 第17回日本遺伝子治療学会
  • Place of Presentation: 九州大学セントラルホール、福岡
  • Year and Date: 2011-07-16
• [Presentation] Stem cell therapy for diabetes mellitus : salivary gland derived progenitors. (2010)
  • Author(s): Shiro Matsumoto
  • Organizer: Japan therapy of gene therapy ; the 16^<th> Annual Meeting 2010
  • Place of Presentation: Tochigi-ken general culture center
  • Year and Date: 2010-07-01
• [Book] 先天代謝異常症ガイドブック (2013)
  • Author(s): 松本志郎、遠藤文夫 著書
  • Publisher: 診断と治療社
• [Book] 先天代謝異常症候群上巻 (2012)
  • Author(s): 松本志郎、遠藤文夫、著書
  • Publisher: 日本臨牀
• [Book] 最先端医療の進歩、膵島再生医療の現状、小児科診療 (2012)
  • Author(s): 松本志郎、遠藤文夫、著書
• [Book] 膵島再生治療の現状 (2012)
  • Author(s): 松本志郎、遠藤文夫
  • Total Pages: 166
  • Publisher: 小児科診療