Nanomedicine has emerged as a promising platform for disease treatment and much progress has been achieved in the clinical translation for cancer treatment. Several types of nanomedicines have been approved for therapeutic application. However, many nanoparticles still suffer from challenges in the translation from bench to bedside. Currently, nanoparticle-based delivery systems have been developed to explore their functions in targeted gene silencing and cancer therapy. This review describes the research progress of different nano-carriers in targeted gene editing, and the recent progress in co-delivery of anticancer drugs and small ribonucleic acid. We also summarize the strategies for improving the specificity of carrier systems. Finally, we discuss the functions of targeted nano-carriers in overcoming chemotherapeutic drug resistance in cancer therapy. As research continues to advance, a better understanding of the safety including long-term toxicity, immunogenicity, and body metabolism may impel nanoparticle translation.