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 |
代理机构 |
|
代理人 |
|
主权项 |
|
地址 |
|