发明名称 THERAPEUTIC MUSCULAR DYSTROPHY DRUG HAVING BUBBLE LIPOSOME LOADED WITH MORPHOLINO AS ACTIVE INGREDIENT
摘要 The present invention addresses the problem of providing a technique for introducing a PMO of markedly improved cell permeability and of considerably improved introduction efficiency. The present invention thereby addresses the problem of providing a therapeutic drug for dramatically mitigating the pathological condition of DMD. The present invention provides a therapeutic drug for Duchenne muscular dystrophy, the drug being characterized in containing as an active ingredient a bubble liposome having a specific morpholino oligomer (PMO) bound to the surface, and characterized in that ultrasonically irradiating muscle tissue from outside the body after administration to muscle tissue or a blood vessel causes the PMO to be introduced with high efficiency into the muscle fiber (muscle cells) of the muscle tissue.
申请公布号 WO2012153635(A1) 申请公布日期 2012.11.15
申请号 WO2012JP61020 申请日期 2012.04.25
申请人 NEPA GENE CO., LTD.;NEGISHI YOICHI;TAKAHASHI YOKO;MARUYAMA KAZUO;ARAMAKI YUKIHIKO 发明人 NEGISHI YOICHI;TAKAHASHI YOKO;MARUYAMA KAZUO;ARAMAKI YUKIHIKO
分类号 A61K47/48;A61K9/127;A61K31/675;A61K48/00;A61P21/04;C12N15/09 主分类号 A61K47/48
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