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Lentiviral vector mediated gene therapy for Krabbe disease

Lentiviral vector mediated gene therapy for Krabbe disease
慢病毒载体介导的克拉伯病基因治疗
批准号:
20591233
负责人:
KOBAYASHI Hiroshi
金额:
$3.0万
依托单位国家:
日本
项目类别:
Grant-in-Aid for Scientific Research (C)
财政年份:
2008
资助国家:
日本
项目状态:
已结题
起止时间:
2008 至 2010

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中文摘要
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英文摘要
We investigated the effect of lenti-virus mediated gene therapy for Krabbe disease. We succeeded in preparing high titer lenti-virus including GALC, deficient enzyme in Krabbe disease expressing gene by large scale harvesting and ultracentrifuge, and injected for neonatal twitcher mouse, model for Krabbe disease resulted in efficient improvement in nutrition and life span. And we had more efficient improvement in the phenotype of twitcher mice by the combination of gene therapy and substrate reduction therapy using L-cycloserine.
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Neonatal Gene Therapy for the mouse model of Krabbe Disease.
克拉伯病小鼠模型的新生儿基因治疗。
DOI: --
发表时间: 2010
期刊:
影响因子: --
作者: [Kobayashi_H., Ariga M., et al.]
通讯作者: et al.
DOI: --
发表时间: 2010
期刊:
影响因子: --
作者: [小林博司, ほか]
通讯作者: ほか
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