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.

证实了通过使用葡糖脑苷脂酶的外观载体精制了真核细胞来源的结合蛋白，并具有酶活性，制备了大量蛋白，并检查了在细胞内外的运动。用生理盐水从小鼠尾静脉短时间给药外观质粒，采用水动力学方法以肝脏为主进行强制外观，并检测酶向血脑屏障的转移。该方法因其在短时间内即可获得高效的外观效果而受到关注。此外，该方法质粒酶外观体内分布证实肽包括各种膜渗透振兴元素联合蛋白质脑移位可能。通过使用能够在静脉中快速给药的流体动力学方法，它成功地使各种修饰的葡萄糖脑苷脂酶-tat酶与肝脏一起出现。当身体运动的内部被评估时，大脑中的变化被检测到，尽管是很小的量。认为其量与其他膜透性肽的量是统一的，有待于进一步研究。

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.