2010 Fiscal Year Final Research Report
Development of mouse model and novel gene therapy for Diamond-Blackfan anemia
| Project/Area Number |
20591265
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| Research Category |
Grant-in-Aid for Scientific Research (C)
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| Allocation Type | Single-year Grants |
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| Section | 一般 |
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| Research Field |
Pediatrics
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| Research Institution | Nippon Medical School |
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Principal Investigator |
MIYAKE Koichi Nippon Medical School, 医学部, 准教授 (90267211)
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| Project Period (FY) |
2008 – 2010
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| Keywords | 貧血 / モデル動物 / RPS19 / 遺伝子治療 / siRNA |
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| Research Abstract |
Diamond-Blackfan anemia (DBA) is a congenital red cell aplasia in which 25% of the patients have a mutation in the ribosomal protein S19 (RPS19) gene. Recently, it was reported that other ribosomal proteins (RPS17 or RPS24 etc.) were also mutated in DBA patients. We constructed lentiviral vector expressing siRNA against RPS17 or RPS24 to analyze the molecular mechanism of DBA. We also constructed an inducible single lentiviral vector, in which both siRNA and KRAB (Kruppel-associated box) gene were contained, and transduced with mouse bone marrow cells or ES cells for development of mouse model of DBA by tetracycline-induced siRNA against RPS17 or RPS24. However, transduction efficiency and cell toxicity of lentiviral vector was one limitation for making mouse model of DBA. Therefore, more development and/or modification of the lentiviral vector may be needed.
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Research Products
(12 results)
