2009 Fiscal Year Final Research Report
Development of gene therapy for SMEI using epilepsy model mouse.
Project/Area Number |
20790759
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Research Category |
Grant-in-Aid for Young Scientists (B)
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Allocation Type | Single-year Grants |
Research Field |
Pediatrics
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Research Institution | The Institute of Physical and Chemical Research |
Principal Investigator |
YAMAGATA Tetsushi The Institute of Physical and Chemical Research, 神経遺伝研究チーム, 研究員 (00338766)
|
Project Period (FY) |
2008 – 2009
|
Keywords | 乳児重症ミオクロニーてんかん / 抗体修飾リポソーム / 遺伝子治療 / SCN1A |
Research Abstract |
In this study, to develop gene therapy for severe myoclonic epilepsy in infancy model mice, pegylated immunoliposomes were investigated and improved. Whereas preparation steps of liposomes have been optimized, expression of transgene was not sufficient to apply the therapy to SMEI model mice. However, results of this study will lead to improvement of liposome that provides enough gene expression in the target tissue. Thus the gene therapy for SMEI model mice may be possible in near future.
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