Experimental study for the treatment of neuronal degeneration in lysosomal storage diseases by modification of autophagy
| Project/Area Number |
23591511
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| Research Category |
Grant-in-Aid for Scientific Research (C)
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| Allocation Type | Multi-year Fund |
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| Section | 一般 |
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| Research Field |
Pediatrics
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| Research Institution | Osaka City University |
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Principal Investigator |
TANAKA Akemi 大阪市立大学, 医学(系)研究科(研究院), 准教授 (30145776)
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| Co-Investigator(Kenkyū-buntansha) |
NAEDA Mitsuyo 公益財団法人先端医療振興財団, 上級研究員 (40122080)
SETO Toshiyuki 大阪市立大学, 大学院医学研究科, 講師 (60423878)
SAWADA Tomo 大阪市立大学, 大学院医学研究科, 研究員 (60585991)
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| Project Period (FY) |
2011 – 2013
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| Project Status |
Completed (Fiscal Year 2013)
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| Budget Amount *help |
¥5,200,000 (Direct Cost: ¥4,000,000、Indirect Cost: ¥1,200,000)
Fiscal Year 2013: ¥1,170,000 (Direct Cost: ¥900,000、Indirect Cost: ¥270,000)
Fiscal Year 2012: ¥1,820,000 (Direct Cost: ¥1,400,000、Indirect Cost: ¥420,000)
Fiscal Year 2011: ¥2,210,000 (Direct Cost: ¥1,700,000、Indirect Cost: ¥510,000)
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| Keywords | ライソゾーム病 / 神経変性 / オートファジー / 経口治療薬 / 神経治療薬 |
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| Research Abstract |
Autophagy is an important factor in the degeneration of neuronal cells of lysosomal storage diseases. We did an experimental study of modifying the autophagy to treat the neurodegeneration in the brain of the mucopolysaccharidosis type II model mouse (IDS-KO).
SCMAS staining of the brain of IDS-KO showed abnormal stimulation of autophagy in various cells especially in neuronal cells, which began from around 6 weeks old IDS-KO. At age 25 weeks, abnormal inclusion bodies appeared in the cytoplasm of neuronal cells of IDS-KO brain, which was not existed at age 20 weeks. We administered chloroquine to IDS-KO orally from 4 weeks old until the examination at 25 weeks old, which findings of the brain were similar to those of 20 weeks old IDS-KO. The result suggested that the inhibition of autophagy could suppress the disease progression in the brain of IDS-KO.
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Report
(4 results)
Research Products
(60 results)
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[Journal Article] Impact of Enzyme Replacement Therapy and Hematopoietic Stem Cell Therapy on Growth in Patients with Hunter Syndrome2014
Author(s)
Patel P, Suzuki Y, Tanaka A, Yabe H, Kato S, Shimada T, Mason RW, Orii KE , Fukao T, Orii T, Tomatsu S
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Journal Title
MGM reports
Volume: 1
Pages: 184-196
Related Report
Peer Reviewed
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[Journal Article] Impact of Enzyme Replacement Therapy and Hematopoietic Stem Cell Therapy on Growth in Patients with Hunter Syndrome2014
Author(s)
Pravin Patel, Yasuyuki Suzuki, Akemi Tanaka, Hiromasa Yabe, Shunichi Kato, Tsutomu Shimada, Robert W. Mason, Kenji E. Orii , Toshiyuki Fukao, Tadao Orii, Shunji Tomatsu
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Journal Title
Molecular Genetics and Metabolism Reports
Volume: 1
Related Report
Peer Reviewed
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[Journal Article] Current diagnosis and management of mucopolysaccharidosis VI in the Asia-Pacific region2012
Author(s)
Hwu WL, Okuyama T, But WM, Estrada S, Gu X, Hui J, Kosuga M, Lin SP, Ngu LH, Shi H, Tanaka A, Thong MK, Wattanasirichaigoon D, Wasant P, McGill J
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Journal Title
Mol. Genet. Metab
Volume: 107
Pages: 136-44
Related Report
Peer Reviewed
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[Journal Article] Long-term efficacy of hematopoietic stem cell transplantation on brain involvement in patients with mucopolysaccharidosis type II : A nationwide survey in Japan2012
Author(s)
Tanaka A, Okuyama T, Suzuki Y, Sakai N, Takakura H, Sawada T, Tanaka T, Otomo T, Ohashi T, Ishige-Wada M, Yabe H, Ohura T, Suzuki N, Kato K, Adachi S, Kobayashi R, Mugishima H, Kato S
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Journal Title
Mol. Genet. Metab
Volume: 107
Pages: 513-20
Related Report
Peer Reviewed
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[Journal Article] Current diagnosis and management of mucopolysaccharidosis VI in the Asia-Pacific region.2012
Author(s)
Hwu WL, Okuyama T, But WM, Estrada S, Gu X, Hui J, Kosuga M, Lin SP, Ngu LH, Shi H, Tanaka A, Thong MK, Wattanasirichaigoon D, Wasant P, McGill J
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Journal Title
Molecular Genetics and Metabolism
Volume: 107
Issue: 1-2
Pages: 136-144
DOI
Related Report
Peer Reviewed
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[Journal Article] Long-term efficacy of hematopoietic stem cell transplantation on brain involvement in patients with mucopolysaccharidosis type II: A nationwide survey in Japan.2012
Author(s)
Tanaka A, Okuyama T, Suzuki Y, Sakai N, Takakura H, Sawada T, Tanaka T, Otomo T, Ohashi T, Ishige-Wada M, Yabe H, Ohura T, Suzuki N, Kato K, Adachi S, Kobayashi R, Mugishima H, Kato S
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Journal Title
Molecular Genetics and Metabolism
Volume: 107
Issue: 3
Pages: 513-520
DOI
Related Report
Peer Reviewed
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[Presentation] Efficacy of Hematopoietic Stem Cell Transplantation versus Enzyme Replacement Therapy on Brain Function in the Patients with Mucopolysaccharidosis Type II2014
Author(s)
22) Tanaka A, Hamazaki T, Okuyama T, Suzuki Y, Sakai N, Kosuga M. Shinpo M, Yabe H, Ishige M, Mugishima H, Tabuchi K, Kato S.
Organizer
10th Annual Symposium of Lysosomal Disease Network
Place of Presentation
Manchester Grand Hyatt, San Diego, U.S.A.
Related Report
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[Presentation] Pseudodeficiency alleleは意外に多く存在する?2013
Author(s)
田中あけみ,濱崎考史,鈴木健,奥山虎之,坂口知子,工藤聡志,門野千穂,藤川研人,小田絵里,藤直子,斎藤三佳,澤田智,北川照,桜庭均,斎藤靜司
Organizer
日本人類遺伝学会第58回大会
Place of Presentation
仙台
Related Report
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[Presentation] Efficacy of enzyme replacement therapy versus hematopoietc stem cell transplantation on brain in MPS II2013
Author(s)
12) Tanaka A, Okuyama T, Suzuki Y, Sakai N, Hamazaki T, Kosuga M. Sawada T, Yabe H, Ishige M, Mugishima H, Kato S.
Organizer
12th International Congres of Inborn Errors of Metabolism
Place of Presentation
Palau de Congressos de Catalunya, Barcelona, Spein
Related Report
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[Presentation] Stabilization of Symptoms and Activity of Daily Living (ADL) in the Patients with Mucopolysaccharidosis Type II Received Hematopoietic Stem Cell Transplantation2012
Author(s)
Tanaka A, Okuyama T, Suzuki Y, Sakai N, Takakura H, Sawada T, Tanaka T, Otomo T, Ohashi T, Wada M, Yabe H, Ohura T, Suzuki N, Kato K, Adachi S, Kobayashi R, Mugishima H, Kato S.
Organizer
12^<th> International Symposium on MPS and Related Diseases
Place of Presentation
Noordwijkerhout, The Netherlands
Related Report
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[Presentation] Stabilization of Symptoms and Activity of Daily Living (ADL) in the Patients with Mucopolysaccharidosis Type II Received Hematopoietic Stem Cell Transplantation.2012
Author(s)
Tanaka A, Okuyama T, Suzuki Y, Sakai N, Takakura H, Sawada T, Tanaka T, Otomo T, Ohashi T, Wada M, Yabe H, Ohura T, Suzuki N, Kato K, Adachi S, Kobayashi R, Mugishima H, Kato S
Organizer
12th International Symposium on MPS and Related Diseases
Place of Presentation
NH Leeuwenhorst Hotel, Noordwijkerhout, The Netherlands
Related Report
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[Presentation] Long-term efficacy of hematopoetic stem cell transplantation in the patients with mucopolysaccharidosis type II : a nationwide survey in Japan2011
Author(s)
Tanaka A, Okuyama T, Suzuki Y, Sakai N, Takakura H, Sawada T, Tanaka T, Otomo T, Ohashi T, Wada M, Yabe H, Ohura T, Suzuki N, Kato K, Adachi S, Kobayashi R, Mugishima H, Kato S.
Organizer
Annual Meeting of Society for the Study of Inborn Errors of Metabolism 2011
Place of Presentation
Geneva, Switzerland
Related Report
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[Presentation] Long-term efficacy of hematopoetic stem cell transplantation in the patients with mucopolysaccharidosis type II: a nationwide survey in Japan2011
Author(s)
Tanaka A, Okuyama T, Suzuki Y, Sakai N, Takakura H, Sawada T, Tanaka T, Otomo T, Ohashi T, Wada M, Yabe H, Ohura T, Suzuki N, Kato K, Adachi S, Kobayashi R, Mugishima H, Kato S
Organizer
Annual Meeting of Society for the Study of Inborn Errors of Metabolism
Place of Presentation
Geneva, Switzerland
Related Report
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[Book] Neurochemistry of Metabolic Disease2012
Author(s)
Tomatsu S, Montaño AM, Molano ACS, Roman D, Hintze J, Carvalho CG, Federhen A, Vieira TA, Giugliani R, Węgrzyn G, Tanaka A, Suzuki Y, Orii T
Publisher
Nova Science Publishers, Inc
Related Report
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[Book] Enzyme Replacement Therapy for Lysosomal Storage Dseases(Neurochemistry of Metabolic Disease)2011
Author(s)
Tomatsu S, Montaño AM, Molano ACS, Roman D, Hintze J, Carvalho CG, Federhen A, Vieira TA, Giugliani R, Węgrzyn G, Tanaka A, Suzuki Y, Orii T. (Chaptor 8)
Publisher
Sanker Surendran, Nova Science Publishers, Inc
Related Report
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