Precision nanomedicine for chemotherapy and gene therapy

Abstract

Precision nanomedicine has emerged as a transformative approach in chemotherapy and gene therapy, leveraging advances in nanotechnology, materials science, and molecular biology to enhance drug delivery efficacy while minimizing off-target toxicity. Conventional chemotherapy faces challenges such as systemic toxicity, drug resistance, and poor tumor specificity, while gene therapy is limited by rapid degradation of genetic payloads (DNA, siRNA, mRNA, CRISPR-Cas9) and inefficient cellular uptake. Nanocarriers, including liposomes, polymeric nanoparticles (PNPs), lipid-based systems, and inorganic nanoparticles, address these limitations by enabling tumor-selective delivery through passive and active targeting, as well as stimuli-responsive release. In chemotherapy, nanoformulations have demonstrated improved pharmacokinetics and reduced side effects, with emerging strategies focusing on codelivery systems and multidrug resistance reversal. For gene therapy, nonviral nanocarriers protect nucleic acids and enhance cellular uptake, endosomal escape, and nuclear localization, enabling CRISPR-based gene editing, RNA interference, and mRNA delivery, exemplified by FDA-approved therapies such as Onpattro. Clinical translation of nanomedicines has seen success with FDA-approved agents and promising candidates in trials, yet challenges remain in scalability, immunogenicity, and personalized adaptation. Future directions include AI-driven nanocarrier design, theranostic systems, and precision oncology approaches tailored to individual tumor profiles. This chapter explores the evolution, mechanisms, clinical applications, and prospects of nanomedicine, highlighting its pivotal role in advancing next-generation cancer therapies.