发明申请
US20140155813A1 THERAPEUTIC MUSCULAR DYSTROPHY DRUG HAVING BUBBLE LIPOSOME LOADED WITH MORPHOLINO AS ACTIVE INGREDIENT 有权
具有作为活性成分的以摩尔作为负载的泡沫脂多糖的治疗性肌肉DYSTROPHY药物

THERAPEUTIC MUSCULAR DYSTROPHY DRUG HAVING BUBBLE LIPOSOME LOADED WITH MORPHOLINO AS ACTIVE INGREDIENT
摘要:
To provide a technology for introducing a PMO with remarkably enhanced cell permeability, leading to significantly enhanced introduction efficiency to thereby provide a therapeutic drug that dramatically ameliorates a medical condition of DMD. Also provided is a therapeutic drug for Duchenne muscular dystrophy, including as an active ingredient a Bubble liposome having a specific morpholino oligomer (PMO) bound to a surface thereof, in which the PMO is introduced into a muscle fiber (muscle cell) of a muscle tissue with high efficiency by administration of the therapeutic drug into the muscle tissue or into a blood vessel followed by ultrasound irradiation to the muscle tissue transcutaneously.
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