Hanes, Justin,Suk, Jung Soo,Mastorakos, Panagiotis,Woodworth, Graeme,Zhang, Clark
申请号:
AU2015259362
公开号:
AU2015259362A1
申请日:
2015.05.12
申请国别(地区):
AU
年份:
2016
代理人:
摘要:
A synthetic gene delivery platform with a dense surface coating of hydrophilic and neutrally charged PEG, capable of rapid diffusion and widespread distribution in brain tissue, and highly effective gene delivery to target cells therein has been developed. Nanoparticles including nucleic acids, are formed of a blend of biocompatible hydrophilic cationic polymers and they hydrophilic cationic polymer conjugated to hydrophilic neutrally charged polymers such as polyethylene glycol. The nanoparticles are coated with polyethylene glycol at a density that imparts a near neutral charge and optimizes rapid diffusion through the brain parenchyma. Methods of treating a disease or disorder of the brain including administering a therapeutically effective amount of nanoparticles densely coated with polyethylene glycol are also provided.