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2009 Fiscal Year Final Research Report

Development of gene therapy for SMEI using epilepsy model mouse.

Research Project

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Project/Area Number 20790759
Research Category

Grant-in-Aid for Young Scientists (B)

Allocation TypeSingle-year Grants
Research Field Pediatrics
Research InstitutionThe 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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Published: 2011-06-18   Modified: 2016-04-21  

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