Pre-Clinical Non-Viral Vectors Exploited for In Vivo CRISPR/Cas9 Gene Editing: An Overview

The review discussed the advancements and challenges of non-viral vectors for in vivo CRISPR/Cas9 gene editing, highlighting their potential in treating diseases like cancer, diabetes, and alopecia. Non-viral vectors, such as lipid nanoparticles (LNPs), polymeric nanoparticles, and cell-penetrating peptides, were explored as safer alternatives to viral vectors, offering reduced immunogenicity and toxicity. Pre-clinical studies demonstrated significant therapeutic outcomes, such as a 67% reduction in tumor growth in melanoma models and up to 90% hair regeneration in androgenetic alopecia by targeting specific genes. Despite promising results, challenges like stability, cellular uptake, and off-target effects remained. The review emphasized the potential of these non-viral strategies to safely and efficiently deliver CRISPR/Cas9 components, paving the way for clinical applications.