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Establishment of gene transfer to hematopoietic cells from patients with congenital neutropenia

Research Project

Project/Area Number 17591093
Research Category

Grant-in-Aid for Scientific Research (C)

Allocation TypeSingle-year Grants
Section一般
Research Field Pediatrics
Research InstitutionKyushu University

Principal Investigator

IHARA Kenji  Kyushu University, Faculty of Medicine, Assistant Professor (80294932)

Co-Investigator(Kenkyū-buntansha) KUSUHARA Koichi  Kyushu University, Faculty of Medicine, Associate Professor (20243941)
曽田 泰  東京大学, 医科学研究所分子療法分野, 助手 (00361618)
Project Period (FY) 2005 – 2007
Project Status Completed (Fiscal Year 2007)
Budget Amount *help
¥3,730,000 (Direct Cost: ¥3,400,000、Indirect Cost: ¥330,000)
Fiscal Year 2007: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
Fiscal Year 2006: ¥900,000 (Direct Cost: ¥900,000)
Fiscal Year 2005: ¥1,400,000 (Direct Cost: ¥1,400,000)
KeywordsRegenerative medicine / Biotechnology / Clinical / Gene / 再生医学
Research Abstract

The study purpose is to establish the gene transfer system and its evaluation method for a growth failure of one of any lineage of hematopoietic progenitors caused by a single gene abnormality We focused on the glycogen storage disease type 1 b associated with congenital neutropenia caused by mutations in glucose 6-translocase (G6PT)gene. First, G6PT gene expression vector
(pCS-CDF-ChG6PT-PRE) was developed. Then CD34-positive cells derived from healthy umbilical cord blood were purified and G6PT gene (or GFP gene) was transferred to the cells by the lentivirus vector. After transfection, methylcellulose colony assay was carried out to evaluate the gene transfer efficiency. Then bone marrow cells from a patient with glycogen storage disease type Ib were collected.G6PT or GFP gene was introduced to the purified CD34-positive cells using the vectors. Methylcellulose colony assay was performed to evaluate the forming ability of G-CFU,GM-CSF,M-CSF and E-BFU colonies. And the cells from each colony were collected and calculated the transduction efficiency by PCR.As a result, gene transfer efficiency was as low as about 10 percent, and gene transfer effect was not significantly improved the forming ability of any type colonies in methylcellulose medium. To find the optimal conditions, this study is now under the way.

Report

(4 results)
  • 2007 Annual Research Report   Final Research Report Summary
  • 2006 Annual Research Report
  • 2005 Annual Research Report
  • Research Products

    (45 results)

All 2008 2007 2006 2005 Other

All Journal Article (28 results) (of which Peer Reviewed: 14 results) Presentation (17 results)

  • [Journal Article] Pulmonary hypertension in patients with congenital portosystemic venous shunt: a previously unrecognized association2008

    • Author(s)
      Ohno T, Ihara K, ほか5名
    • Journal Title

      Pediatrics 121

    • Description
      「研究成果報告書概要(和文)」より
    • Related Report
      2007 Final Research Report Summary
    • Peer Reviewed
  • [Journal Article] Genetic analysis of MMP gene polymorphisms in patients with Kawasaki disease2008

    • Author(s)
      Ikeda K, Ihara K, ほか5名
    • Journal Title

      Pediatric Research Feb;63(2)

      Pages: 182-5

    • Description
      「研究成果報告書概要(和文)」より
    • Related Report
      2007 Annual Research Report 2007 Final Research Report Summary
    • Peer Reviewed
  • [Journal Article] PD-1 gene haplotype is associated with the development of type 1 diabetes mellitus in Japanese children2007

    • Author(s)
      Ni R、Ihara K, ほか5名
    • Journal Title

      Human Genetics 121(2)

      Pages: 223-232

    • Description
      「研究成果報告書概要(和文)」より
    • Related Report
      2007 Final Research Report Summary
    • Peer Reviewed
  • [Journal Article] The Leu544Ile Polymorphism of the growth hormone receptor gene affects the serum cholesterol levels during GH treatment in children with GH deficiency2007

    • Author(s)
      Ihara K, ほか6名
    • Journal Title

      Clinical Endocrinology 67

      Pages: 212-217

    • NAID

      40016612565

    • Description
      「研究成果報告書概要(和文)」より
    • Related Report
      2007 Annual Research Report 2007 Final Research Report Summary
    • Peer Reviewed
  • [Journal Article] Novel mutations in five Japanese patients with 3-methylcrotony1-CoA carboxylase deficiency2007

    • Author(s)
      Uematsu M, Ihara K, ほか16名
    • Journal Title

      Journal of Human Genetics 52(12)

      Pages: 1040-1043

    • Description
      「研究成果報告書概要(和文)」より
    • Related Report
      2007 Final Research Report Summary
    • Peer Reviewed
  • [Journal Article] PD-1 gene haplotype is associated with the development of type 1 diabetes mellitus in Japanese children2007

    • Author(s)
      Ni, R., Ihara, K., Miyako, K., Kuromaru, R., Inuo, M., Kohno, H., Hara., T
    • Journal Title

      Hum Genet. 121(2)

      Pages: 223-32

    • Description
      「研究成果報告書概要(欧文)」より
    • Related Report
      2007 Final Research Report Summary
  • [Journal Article] The Leu544Ile polymorphism of the growth hormone receptor gene affects the serum cholesterol levels during GH treatment in children with GH deficiency2007

    • Author(s)
      Ihara, K., Inuo, M., Kuromaru, R., Miyako, K., Kohno, H., Kinukawa, N., Hara., T
    • Journal Title

      Clin Endocrinol. 67

      Pages: 212-217

    • NAID

      40016612565

    • Description
      「研究成果報告書概要(欧文)」より
    • Related Report
      2007 Final Research Report Summary
  • [Journal Article] Novel mutations in five Japanese patients with 3-methylcrotonyl-CoA carboxylase deficiency2007

    • Author(s)
      Uematsu, M., Sakamoto, O., Sugawara, N., Kumagai, N., Morimoto, T., Yamaguchi, S., Hasegawa, Y., Kobayashi, H., Ihara, K, Yoshino, M., Watanabe, Y., Inokuchi, T., Yokoyama, T., Kiwaki, K., Nakamura, K., Endo, F., Tsuchiya, S., Ohura, T
    • Journal Title

      J Hum Genet. 52(12)

      Pages: 1040-3

    • NAID

      10020022418

    • Description
      「研究成果報告書概要(欧文)」より
    • Related Report
      2007 Final Research Report Summary
  • [Journal Article] PD-1 gene haplotype is associated with the development of type 1 diabetes mellitus in Japanese children2007

    • Author(s)
      Ni R、Ihara K ほか5名
    • Journal Title

      Human Genetics 121(2)

      Pages: 223-232

    • Related Report
      2007 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Novel mutations in five Japanese patients with 3-methylcrotonyl-CoA carboxylase deficiency2007

    • Author(s)
      Uematsu M, Ihara K, ほか16名
    • Journal Title

      Journal of Human Genetics 52(12)

      Pages: 1040-1043

    • NAID

      10020022418

    • Related Report
      2007 Annual Research Report
    • Peer Reviewed
  • [Journal Article] PD-1 gene haplotype is associated with the development of type 1 diabetes mellitus in Japanese children.2007

    • Author(s)
      Ni Rほか6名
    • Journal Title

      Human Genetics 121(2)

      Pages: 223-232

    • Related Report
      2006 Annual Research Report
  • [Journal Article] Association study of polymorphisms in SOCS family genes with type 1 diabetes mellitus2006

    • Author(s)
      Ni R, Ihara K, ほか7名
    • Journal Title

      Int J Immunogenet 33(1)

      Pages: 7-10

    • Description
      「研究成果報告書概要(和文)」より
    • Related Report
      2007 Final Research Report Summary
    • Peer Reviewed
  • [Journal Article] Phenotypic and genetic analyses of T cell-mediated immunoregulation in patients with type 1 diabetes2006

    • Author(s)
      Tsutsumi Y, Ihara K, ほか5名
    • Journal Title

      Diabetic Med 23(10)

      Pages: 1145-50

    • Description
      「研究成果報告書概要(和文)」より
    • Related Report
      2007 Final Research Report Summary
    • Peer Reviewed
  • [Journal Article] Persistent diffusion abnormalities in the brain stem of three children with mitochondrial diseases2006

    • Author(s)
      Sakai Y, Ihara K, ほか4名
    • Journal Title

      Am J Neuroradiol 27(9)

      Pages: 1924-6

    • Description
      「研究成果報告書概要(和文)」より
    • Related Report
      2007 Final Research Report Summary
    • Peer Reviewed
  • [Journal Article] Association study of polymorphisms in SOCS family genes with type 1 diabetes mellitus2006

    • Author(s)
      Ni, R., Ihara, K., Miyako, Takemoto, M, Ishimura, M., Kohno, H., Matsuura, N., Yoshimura, A., Hara, T
    • Journal Title

      Int J Immunogenet 33(1)

      Pages: 7-10

    • Description
      「研究成果報告書概要(欧文)」より
    • Related Report
      2007 Final Research Report Summary
  • [Journal Article] Phenotypic and genetic analyses of T cell-mediated immunoregulation in patients with type 1 diabetes2006

    • Author(s)
      Tsutsumi, Y., Jie, X., Ihara, K., Nomura, A., Kanemitsu, S., Takada, H., Hara, T
    • Journal Title

      Diabetic Med. 23(10)

      Pages: 1145-50

    • Description
      「研究成果報告書概要(欧文)」より
    • Related Report
      2007 Final Research Report Summary
  • [Journal Article] Persistent diffusion abnormalities in the brain stem of three children with mitochondrial diseases2006

    • Author(s)
      Sakai, Y., Kira, R., Torisu, H., Ihara, K., Yoshiura, T., Hara, T
    • Journal Title

      Am J Neuroradiol. 27(9)

      Pages: 1924-6

    • Description
      「研究成果報告書概要(欧文)」より
    • Related Report
      2007 Final Research Report Summary
  • [Journal Article] Association study of polymorphisms in SOCS family genes with type 1 diabetes mellitus.2006

    • Author(s)
      Ni Rほか8名
    • Journal Title

      International Journal of Immunogenetics 33(1)

      Pages: 7-10

    • Related Report
      2005 Annual Research Report
  • [Journal Article] Successful bone marrow transplantation in a patient with c-mpl-mutated congenital amegakaryocytic thrombocytopenia from a carrier donor2005

    • Author(s)
      Muraoka K, Ihara K, ほか5名
    • Journal Title

      Pediatr Transplant 9(1)

      Pages: 101-103

    • Description
      「研究成果報告書概要(和文)」より
    • Related Report
      2007 Final Research Report Summary
    • Peer Reviewed
  • [Journal Article] Thrombocytosis in Preterm Infants: A Possible Involvelnent of Thrombopo ietin Receptor Gene Expression2005

    • Author(s)
      Nakayama H, Ihara K, ほか5名
    • Journal Title

      J Mol Med 83(4)

      Pages: 316-320

    • Description
      「研究成果報告書概要(和文)」より
    • Related Report
      2007 Final Research Report Summary
    • Peer Reviewed
  • [Journal Article] A case of hyperinsulinism/hyperammonemia syndrome with reduced carbamy Iphosphate synthetase-1 activity in liver: a pitfall in enzymatic diagnosis for hyperammonemia2005

    • Author(s)
      Ihara K, ほか6名
    • Journal Title

      J Inherit Metab Dis 28(5)

      Pages: 681-687

    • Description
      「研究成果報告書概要(和文)」より
    • Related Report
      2007 Final Research Report Summary
    • Peer Reviewed
  • [Journal Article] Congenital Amegakaryocytic Thrombocytopenia in Three Siblings: Molecular Analysis of Atypical Clinical Presentation2005

    • Author(s)
      Gandhi MJ, Ihara K, ほか4名
    • Journal Title

      Exp Hematol 33

      Pages: 1215-1221

    • Description
      「研究成果報告書概要(和文)」より
    • Related Report
      2007 Final Research Report Summary
    • Peer Reviewed
  • [Journal Article] Successful bone marrow transplantation in a patient with c-mpl-mutated congenital amegakaryocytic thrombocytopenia from a carrier donor2005

    • Author(s)
      Muraoka, K., Ishii, E., Ihara, K., Imayoshi, M., Miyazaki, S., Hara, T., Hamasaki, Y
    • Journal Title

      Pediatr Transplant 9(1)

      Pages: 101-103

    • Description
      「研究成果報告書概要(欧文)」より
    • Related Report
      2007 Final Research Report Summary
  • [Journal Article] Thrombocytosis in Preterm Infants : A Possible Involvement of Thrombopoietin Receptor Gene Expression2005

    • Author(s)
      Nakayama, H., Ihara, K., Hikino, S., Yamamoto, J., Nagatomo, T., Takemoto, M., Hara, T
    • Journal Title

      J Mol Med 83(4)

      Pages: 316-320

    • Description
      「研究成果報告書概要(欧文)」より
    • Related Report
      2007 Final Research Report Summary
  • [Journal Article] A case of hyperinsulinism/hyperammonemia syndrome with reduced carbamylphosphate synthetase-1 activity in liver : a pitfall in enzymatic diagnosis for hyperammonemia2005

    • Author(s)
      Ihara, K., Miyako, K., Ishimura, M., Kuromaru, R., Wang, H-Y., Yasuda, K., Hara, T
    • Journal Title

      J Inherit Metab Dis 28(5)

      Pages: 681-687

    • Description
      「研究成果報告書概要(欧文)」より
    • Related Report
      2007 Final Research Report Summary
  • [Journal Article] Congenital Amegakaryocytic Thrombocytopenia in Three Siblings : Molecular Analysis of Atypical Clinical Presentation2005

    • Author(s)
      Gandhi, MJ., Pendergrass, TW., Cummings, CC., Ihara, K., Blau, CA., Drachman, JG
    • Journal Title

      Exp Hematol 33

      Pages: 1215-1221

    • Description
      「研究成果報告書概要(欧文)」より
    • Related Report
      2007 Final Research Report Summary
  • [Journal Article] Genetic analysis of MMP gene polymorphisms in patients with Kawasaki disease

    • Author(s)
      Ikeda, K., Ihara, K., Yamaguchi, K., Muneuchi, J., Ohno, T., Mizuno, Y., Hara, T
    • Journal Title

      Pediatr Res. (in press)

    • Description
      「研究成果報告書概要(欧文)」より
    • Related Report
      2007 Final Research Report Summary
  • [Journal Article] Pulmonary hypertension in patients with congenital porto-systemic venous shunt : a previously unrecognized association

    • Author(s)
      Ohno, T., Muneuchi, J., Ihara, K., Yuge, T., Kanaya, Y., Yamaki, S., Hara, T
    • Journal Title

      Pediatrics. (in press)

    • Description
      「研究成果報告書概要(欧文)」より
    • Related Report
      2007 Final Research Report Summary
  • [Presentation] スルフォニルウレア剤内服治療によりインスリン治療離脱が可能であった、Kir6.2遺伝子異常による新生児糖尿病の1男児例2007

    • Author(s)
      高柴 朋子、井原 健二, ほか3名
    • Organizer
      第7回日本内分泌学会九州地方会
    • Place of Presentation
      福岡
    • Year and Date
      2007-09-01
    • Description
      「研究成果報告書概要(和文)」より
    • Related Report
      2007 Final Research Report Summary
  • [Presentation] A case of infant diabetes with a mutation in the Kir6.2 gene : improvement by sulfonylurea treatment2007

    • Author(s)
      Takashiba, T., Ihara, K., et. al.
    • Organizer
      7th Annual Meeting of Japanese Society of Endocrinology in Kyushu
    • Place of Presentation
      Fukuoka, Japan
    • Year and Date
      2007-09-01
    • Description
      「研究成果報告書概要(欧文)」より
    • Related Report
      2007 Final Research Report Summary
  • [Presentation] 成長ホルモン治療による治療感受性を規定する遺伝学的背景因子〜成長ホルモン受容体遺伝子多型の解析〜2007

    • Author(s)
      井原 健二
    • Organizer
      KIGSフォーラムin福岡
    • Place of Presentation
      福岡
    • Year and Date
      2007-03-17
    • Description
      「研究成果報告書概要(和文)」より
    • Related Report
      2007 Final Research Report Summary
  • [Presentation] Polymorphisms in the growth hormone receptor for growth response and disease susceptibility2007

    • Author(s)
      Ihara, K
    • Organizer
      KIGS forum in Fukuoka
    • Place of Presentation
      Fukuoka, Japan
    • Year and Date
      2007-03-17
    • Description
      「研究成果報告書概要(欧文)」より
    • Related Report
      2007 Final Research Report Summary
  • [Presentation] 成長ホルモン治療による脂質代謝に関与する成長ホルモン受容体遺伝子多型の解析2007

    • Author(s)
      井原 健二, ほか5名
    • Organizer
      第443回日本小児科学会福岡地方会
    • Place of Presentation
      福岡
    • Year and Date
      2007-02-10
    • Description
      「研究成果報告書概要(和文)」より
    • Related Report
      2007 Final Research Report Summary
  • [Presentation] The Leu544Ile Polymorphism of the Growth Hormone Receptor Gene Affects the Serum Cholesterol Levels during GH treatment in Children with GH Deficiency2007

    • Author(s)
      Ihara, K., Inuo, M., Kuromaru, R., Miyako, K., Kohno, Hi., Hara, T
    • Organizer
      433rd Meeting of the Japanese Pediatric Society in Fukuoka Prefecture
    • Place of Presentation
      Fukuoka, Japan
    • Year and Date
      2007-02-10
    • Description
      「研究成果報告書概要(欧文)」より
    • Related Report
      2007 Final Research Report Summary
  • [Presentation] The Leu544Ile Polymorphism of the Growth Hormone Receptor Gene Affects the Serum Cholesterol Levels during GH treatment in Children with GH Deficiency2007

    • Author(s)
      Ihara K, ほか6名
    • Organizer
      3rd Congress of the Asian Society of Pediatric Research
    • Place of Presentation
      Tokyo,Japan
    • Description
      「研究成果報告書概要(和文)」より
    • Related Report
      2007 Annual Research Report 2007 Final Research Report Summary
  • [Presentation] Possibility of Congenital Porto-systemic Venous Shunt as Latent Causeg in Pulmonary Arterial Hypertension: A Case Study of Clinical Manifestation and Outcome2007

    • Author(s)
      Ohno T, Ihara K, ほか5名
    • Organizer
      3rd Congress of the Asian Society of Pediatric Research
    • Place of Presentation
      Tokyo,Japan
    • Description
      「研究成果報告書概要(和文)」より
    • Related Report
      2007 Final Research Report Summary
  • [Presentation] 成長ホルモン治療による脂質代謝に関与する成長ホルモン受容体遺伝子多型の解析2007

    • Author(s)
      井原 健二, ほか5名
    • Organizer
      第52回日本人類伝学会
    • Place of Presentation
      東京
    • Description
      「研究成果報告書概要(和文)」より
    • Related Report
      2007 Annual Research Report 2007 Final Research Report Summary
  • [Presentation] 成長ホルモン治療による脂質代謝に関与する成長ホルモン受容体遺伝子多型の解析2007

    • Author(s)
      井原 健二, ほか5名
    • Organizer
      第41回日本小児内分泌学会
    • Place of Presentation
      横浜
    • Description
      「研究成果報告書概要(和文)」より
    • Related Report
      2007 Final Research Report Summary
  • [Presentation] 成長ホルモン治療反応性に関わる遺伝子多型解析(2)2007

    • Author(s)
      犬尾 美佳、井原 健二, ほか4名
    • Organizer
      第41回日本小児内分泌学会
    • Place of Presentation
      横浜
    • Description
      「研究成果報告書概要(和文)」より
    • Related Report
      2007 Final Research Report Summary
  • [Presentation] The Leu544Ile Polymorphism of the Growth Hormone Receptor Gene Affects the Serum Cholesterol Levels during GH treatment in Children with GH Deficiency2007

    • Author(s)
      Ihara, K., Inuo, M., Kuromaru, R., Miyako, K., Kohno, Hi., Hara, T
    • Organizer
      3rd Congress of the Asian Society of Pediatric Research
    • Place of Presentation
      Tokyo, Japan
    • Description
      「研究成果報告書概要(欧文)」より
    • Related Report
      2007 Final Research Report Summary
  • [Presentation] Possibility of Congenital Porto-systemic Venous Shunt as Latent Cause in Pulmonary Arterial Hypertension : A Case Study of Clinical Manifestation and Outcome2007

    • Author(s)
      Ohno, T., Muneuchi, J., Ihara, K., Yuge, T., Kanaya, Y., Yamaki, S., Hara, T
    • Organizer
      3rd Congress of the Asian Society of Pediatric Research
    • Place of Presentation
      Tokyo, Japan
    • Description
      「研究成果報告書概要(欧文)」より
    • Related Report
      2007 Final Research Report Summary
  • [Presentation] The Leu544Ile Polymorphism of the Growth Hormone Receptor Gene Affects the Serum Cholesterol Levels during GH treatment in Children with GH Deficiency2007

    • Author(s)
      Ihara, K., Inuo, M., Kuromaru, R., Miyako, K., Kohno, Hi., Hara, T
    • Organizer
      52nd Annual Meeting of Japanese Society of Human Genetics
    • Place of Presentation
      Tokyo, Japan
    • Description
      「研究成果報告書概要(欧文)」より
    • Related Report
      2007 Final Research Report Summary
  • [Presentation] The Leu544Ile Polymorphism of the Growth Hormone Receptor Gene Affects the Serum Cholesterol Levels during GH treatment in Children with GH Deficiency2007

    • Author(s)
      Ihara, K., Inuo, M., Kuromaru, R., Miyako, K., Kohno, Hi., Hara, T
    • Organizer
      41st Annual Meeting of Japanese Society of Pediatric Endocrinology
    • Place of Presentation
      Yokohama, Japan
    • Description
      「研究成果報告書概要(欧文)」より
    • Related Report
      2007 Final Research Report Summary
  • [Presentation] Polymorphisms in the growth hormone receptor for growth response(2)2007

    • Author(s)
      Inuo, M., Ihara, K., et. al.
    • Organizer
      41st Annual Meeting of Japanese Society of Pediatric Endocrinology
    • Place of Presentation
      Yokohama, Japan
    • Description
      「研究成果報告書概要(欧文)」より
    • Related Report
      2007 Final Research Report Summary
  • [Presentation] 成長ホルモン治療による脂質代謝に関与する成長ホルモン受容体遺伝子多型の解析2007

    • Author(s)
      井原 健二, ほか5名
    • Organizer
      第110回日本小児科学会
    • Place of Presentation
      京都
    • Related Report
      2007 Annual Research Report

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Published: 2005-04-01   Modified: 2016-04-21  

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