Drug screening for Diamond-Blackfan anemia using zebrafish as a model animal
| Project/Area Number |
15K15367
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| Research Category |
Grant-in-Aid for Challenging Exploratory Research
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| Allocation Type | Multi-year Fund |
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| Research Field |
Hematology
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| Research Institution | University of Miyazaki |
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Principal Investigator |
Kenmochi Naoya 宮崎大学, フロンティア科学実験総合センター, 教授 (00133124)
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| Project Period (FY) |
2015-04-01 – 2018-03-31
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| Project Status |
Completed (Fiscal Year 2017)
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| Budget Amount *help |
¥3,640,000 (Direct Cost: ¥2,800,000、Indirect Cost: ¥840,000)
Fiscal Year 2016: ¥1,820,000 (Direct Cost: ¥1,400,000、Indirect Cost: ¥420,000)
Fiscal Year 2015: ¥1,820,000 (Direct Cost: ¥1,400,000、Indirect Cost: ¥420,000)
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| Keywords | ダイアモンド・ブラックファン貧血 / 先天性貧血 / リボソーム / 化合物ライブラリー / 創薬 / ゼブラフィッシュ / 疾患モデル / 化合物スクリーニング / ダイヤモンド・ブラックファン貧血 / 造血異常 / 低分子化合物 / リボソーム病 |
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| Outline of Final Research Achievements |
Diamond-Blackfan anemia (DBA) is a congenital bone marrow failure syndrome characterized by red blood cell aplasia. Although the ribosomal dysfunction is implicated in this disease, the molecular pathogenesis remains unknown, thereby no effective treatment exists. In this study, we performed in vivo screening of 1,280 small compounds using zebrafish to identify the drug candidates for DBA. We found that four compounds effectively rescued the anemia phenotype in zebrafish, suggesting that these are the promising pharmaceutical seed materials for DBA treatment.
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Report
(4 results)
Research Products
(39 results)
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[Journal Article] Identification and characterization of 5alpha-cyprinol-sulfating cytosolic sulfotransferases (Sults) in the Zebrafish (Danio rerio).2017
Author(s)
Kurogi, K., Yoshihama, M., Horton, A., Schiefer, I.T., Krasowski, M.D., Hagey, L.R., Williams, F.E., Sakakibara, Y., Kenmochi, N., Suiko, M., Liu, M.-C.
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Journal Title
The Journal of Steroid Biochemistry and Molecular Biology
Volume: 174
Pages: 120-127
DOI
Related Report
Peer Reviewed / Open Access / Int'l Joint Research
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[Journal Article] TBCD may be a causal gene in progressive neurodegenerative encephalopathy with atypical infantile spinal muscular atrophy2017
Author(s)
Ikeda T, Nakahara A, Nagano R, Utoyama M, Obara M, Moritake H, Uechi T, Mitsui J, Ishiura H, Yoshimura J, Doi K, Kenmochi N, Morishita S, Nishino I, Tsuji S, and Nunoi H
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Journal Title
J Hum Genet
Volume: 62
Issue: 4
Pages: 473-480
DOI
NAID
Related Report
Peer Reviewed / Open Access
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[Journal Article] Exome sequencing identified RPS15A as a novel causative gene for Diamond-Blackfan anemia.2017
Author(s)
Ikeda F, Yoshida K, Toki T, Uechi T, Ishida S, Nakajima Y, Sasahara Y, Okuno Y, Kanezaki R, Terui K, Kamio T, Kobayashi A, Fujita T, Sato-Otsubo A, Shiraishi Y, Tanaka H, Chiba K, Muramatsu H, Kanno H, Ohga S, Ohara A, Kojima S, Kenmochi N, Miyano S, Ogawa S, Ito E.
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Journal Title
Haematologica.
Volume: -
Issue: 3
Pages: e93-e96
DOI
Related Report
Peer Reviewed / Open Access / Acknowledgement Compliant
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