Methods of the early detection of diseases are based on recognition of the smallest change in the levels of a disease-specific biomarker in body fluids.
MicroRNA (miR), a key molecule involved in endogenous RNA interference, is a promising therapeutic agent. In vivo delivery of miR, however, is a major factor limiting its application because its polyanionic nature and vulnerability to breakdown make delivery of miR to targeted lesions difficult. To overcome these challenges, we developed a self-assembled miR delivery system consisting of cholesterol-conjugated miR and polyethylene glycol-grafted polyethylene imine. Nanosized complexes of miR with polyethylene imine, which protected miR and its delivery into targeted lesions in vivo, were successfully synthesized by polyethylene glycol grafting. The hydrophobicity of cholesterol improved the structural stability of the complex, preventing the loss of miR. Here, we report the preparation of this self-assembled complex. We examined the delivery efficiency and validated the therapeutic efficacy of the complex. In conclusion, our miR delivery system shows considerable potential for effective in vivo delivery of miR.
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