Development of the safe gene therapy method for single gene disorder without insertion to a host genom

开发无需插入宿主基因组的单基因疾病安全基因治疗方法

• 批准号: 23790602
• 负责人: YOSHIDA Kumi
• 金额: $ 1.41万
• 依托单位: Kyushu University
• 依托单位国家: 日本
• 项目类别: Grant-in-Aid for Young Scientists (B)
• 财政年份: 2011
• 资助国家: 日本
• 起止时间: 2011 至 2012
• 项目状态: 已结题
• 来源: https://kaken.nii.ac.jp/grant/KAKENHI-PROJECT-23790602/
• 关键词: 遺伝子治療; 単一遺伝子欠損症

Ｗe had reported that in vivo repopulation of cytoplasmically gene transferred hematopoietic cells by tsSeV/dF-GFP. But, it has not reached in semipermanent maintenance of transgenics protein.Then, to notice the self renewal of hematopoietic stem cells. We investigated how rSeV influenced to reactive oxygen species and the dynamic state of Ca2+ in vivo. As a result, long -term survival and maintaining of transgene semipermanently was not only by decreasing reactive oxygen species with an antioxidant agent. Change of the hormone relevant to Ca2+ and Ca2+ in the gene transferred bone marrow transplant mouse by tsSeV/dF-GFP was seen.In maintenance of semipermanent of the gene transferred by tsSeV/dF-GFP , the relation of not only reactive oxygen species but also Ca2+ was suggested deeply.

我们报告说，TSSEV/DF-GFP的细胞质转移了造血细胞。在体内，作为抗氧化剂的反应性氧与Ca2+相关的抗氧化剂的变化是通过TSSEV/DF-GFP中的Ca2+降低与Ca2+相关的激素的变化，从而降低了反应性氧气。由TSSEV/DF-GFP F转移不仅是活性氧，而且还建议Ca2+转移。