Current Issues

The advent of clustered regularly interspaced short palindromic repeats (CRISPR)/Cas systems has revolutionized the field of gene therapy, offering unparalleled precision, programmability, and efficiency for genome editing. However, translating CRISPR-based therapeutics into clinical applications remains constrained by major challenges in the safe and targeted delivery of the CRISPR components—Cas nucleases and guide RNAs—into specific cells and tissues. Recent advances in nanotechnology have provided innovative solutions to these limitations, leading to the emergence of CRISPR-based nanotherapeutics. Nanocarriers such as lipid nanoparticles, polymeric systems, inorganic nanoparticles, and biomimetic exosomes enable protection of CRISPR cargos from enzymatic degradation, facilitate endosomal escape, and improve tissue-specific targeting while minimizing off-target effects and immunogenicity. This review comprehensively discusses the principles and mechanisms of CRISPR/Cas systems, various nanoplatforms engineered for their delivery, and design strategies for optimizing safety and efficacy. Special emphasis is given to the applications of CRISPR–nanocarrier combinations in cancer therapy, genetic and infectious diseases, neurological disorders, and regenerative medicine. Furthermore, recent preclinical progress, ongoing clinical trials, regulatory considerations, and future perspectives are highlighted. The convergence of genome editing and nanotechnology represents a transformative step toward next-generation, personalized, and precision gene therapies with potential to redefine the future of molecular medicine.

Keyword: CRISPR/Cas9, Nanoparticle, Gene editing, Targeted delivery, Personalized medicine, Genome engineering