Rod-Shaped Active Drug Particles Enable Efficient and Safe Gene Delivery.

Efficient microRNAs (miRNA) delivery into cells is a promising strategy for disease therapy, but is a major challenge because the available conventional nonviral vectors have significant drawbacks. In particular, after these vectors are entrapped in lysosomes, the escape efficiency of genes from lysosomes into the cytosol is less than 2%. Here, a novel approach for lethal-7a (let-7a) replacement therapy using rod-shaped active pure drug nanoparticles (≈130 nm in length, PNPs) with a dramatically high drug-loading of ≈300% as vectors is reported.