Lipid Nanocarriers as a Novel Platform for Precision Delivery of CRISPR-Based Gene Editing Systems and RNA Therapy in Glioblastoma

Abstract

Glioblastoma (GBM) is one of the most aggressive and lethal brain tumors, characterized by high heterogeneity, rapid progression, and resistance to current therapeutic strategies. Standard treatments—including surgery, radiotherapy, and temozolomide chemotherapy—have limited efficacy, and median survival remains approximately 15 months. Among various molecular drivers of GBM, microRNA-10b (miR-10b) plays a central oncogenic role, being consistently upregulated in glioma cells while absent in normal brain tissue. Its inhibition demonstrates strong therapeutic potential. However, a major challenge is the efficient and safe delivery of gene-editing systems to intracranial tumors. Recent advancements in lipid nanoparticles (LNPs) have made them a promising platform for delivering mRNA and sgRNA for CRISPR-based therapies.