发明名称 THERAPEUTIC MUSCULAR DYSTROPHY DRUG HAVING BUBBLE LIPOSOME LOADED WITH MORPHOLINO AS ACTIVE INGREDIENT
摘要 <P>PROBLEM TO BE SOLVED: To provide a technique for introducing a PMO (morpholino oligomer) of markedly improved cell permeability and of considerably improved introduction efficiency, and thereby to provide a therapeutic drug for dramatically mitigating the pathological condition of DMD (Duchenne muscular dystrophy). <P>SOLUTION: There is provided the therapeutic drug for DMD characterized by containing as an active ingredient a bubble liposome having a specific morpholino oligomer (PMO) bound to the surface, and further characterized in that the PMO is introduced with high efficiency to muscle fiber (muscle cells) of a muscle tissue by irradiating the muscle tissue with supersonic waves from the outside of the body after administration to the muscle tissue or a blood vessel. <P>COPYRIGHT: (C)2013,JPO&INPIT
申请公布号 JP2012232957(A) 申请公布日期 2012.11.29
申请号 JP20110104300 申请日期 2011.05.09
申请人 TOKYO UNIV OF PHARMACY & LIFE SCIENCE;NEPA GENE CO LTD 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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