发明授权
US09457102B2 Therapeutic muscular dystrophy drug having bubble liposome loaded with morpholino as active ingredient 有权
具有负载吗啉代作为活性成分的气泡脂质体的治疗性肌营养不良药物

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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