Analysis of shift in brain with film penetration peptide uniting enzyme for nerve type Gaucher disease treatment

透膜肽联合酶治疗神经型戈谢病脑转移分析

• 批准号: 15591141
• 负责人: WATANABE Atsushi
• 金额: $ 1.92万
• 依托单位: Nippon Medical School
• 依托单位国家: 日本
• 项目类别: Grant-in-Aid for Scientific Research (C)
• 财政年份: 2003
• 资助国家: 日本
• 起止时间: 2003 至 2004
• 项目状态: 已结题
• 来源: https://kaken.nii.ac.jp/en/grant/KAKENHI-PROJECT-15591141/
• 关键词: Gaucher diseas; glucocerebrosidase; penetration peptide uniting enzyme; 神経型Gaucher病; 膜透過ペプチド; 血液脳関門; TAT融合蛋白

It is confirmed to refine the uniting protein of the eukaryocyte origin by using the appearance vector for glucocerebrosidase, and to have the enzymic activity, and a large amount of protein is made and the movement in the inside and outside of the cell is examined. The appearance plasmid is administered from mouse's coccygeal vein for a short time with the physiologic saline, the Hydrodynamic method to do the compulsion appearance by the liver chiefly is used, and the shift of the enzyme to the blood brain barrier is examined. This method is paid to attention recently because highly effective appearance is seen in easy and a short time. In addition, this.method.plasmid.enzyme.appearance.invivo.distribution.confirm.peptide.including.various.film.penetration.revitalization.element.unite.protein.brain.shift.possible. It succeeded in strong making various modification glucocerebrosidase-tat enzymes appear with the liver by using the Hydrodynamics method that was able to be administered rapidly in the vein. When the inside of the body movement had been evaluated, the shift in the brain was detected though was a small amount. It was thought that the amount and other film penetration peptide were united and a further examination was necessary in the future.

可以通过使用外观载体进行葡萄糖脑苷酶，并具有大量蛋白质，并检查细胞内部和外部的运动。外观质粒是通过生理盐水在短时间内从小鼠的尾静脉施用的，主要使用肝脏的水动力学方法，主要使用肝脏的强迫性外观，并检查了酶向血脑屏障的转移。由于在轻松且短时间内可以看到高效的外观，因此该方法受到关注。此外，this.method.plasmid.enzyme.appearance.invivo.distribution.confirm.peptide.including.various.film.penetration.revitalization.revitalization.element.unite.unite.protein.protein.brain.brain.brain.shift.popsible.poppible。它通过使用能够在静脉中迅速施用的流体动力学方法来成功使各种修饰葡萄糖葡萄糖酶溴酶-TAT酶出现在肝脏中。当对身体运动的内部运动进行了评估时，检测到大脑的转移量很小。据认为，数量和其他膜穿透肽是团结的，将来需要进一步检查。

项目成果

Migita M., Hamada, H., Fujimura, J., Watanabe, A., Shimada, T., Fukunaga.Y.: "Glucocerebrosidase level in the cerebrospinal fluid during enzymerepla cement therapy -unsuccessful treatment to the neurological abnormality of type 2 Gaucher disease-."Eur.J.P

Migita M.、Hamada, H.、Fujimura, J.、Watanabe, A.、Shimada, T.、Fukunaga.Y.：“酶替代水泥治疗期间脑脊液中的葡萄糖脑苷脂酶水平 - 对 2 型神经异常的治疗失败

modification of the expression vector for systemic gene therapy of MLD

MLD全身基因治疗表达载体的修饰

• 发表时间: 2004
• 期刊: Molecular Therapy 9Supple
• 作者: Ito A;Uno T;Gunji Y;et al.;Takakusaki S et al.
• 通讯作者: Takakusaki S et al.