Gene Therapy: Advances in Delivery Systems, Genome Editing Technologies, and Applications in Human and Veterinary Medicine

Gene therapy has emerged as a revolutionary therapeutic strategy for the treatment of inherited, acquired, and age-related diseases through the modification, replacement, or correction of defective genes. Advances in molecular biology, viral engineering, and genome-editing technologies have significantly enhanced the efficiency and safety of gene delivery systems. This review provides a comprehensive overview of gene therapy, including its classification, delivery methods, and therapeutic applications in both human and veterinary medicine. Viral vectors such as adenoviral, retroviral, lentiviral, adeno-associated viral, and poxviral vectors remain the most effective delivery platforms owing to their high transduction efficiency, whereas non-viral approaches including physical and chemical delivery systems offer improved biosafety and reduced immunogenicity. Recent developments in gene-editing technologies, particularly clustered regularly interspaced short palindromic repeats (CRISPR)-associated protein systems, have expanded the therapeutic potential of gene therapy by enabling precise genome modifications. The review highlights the successful application of gene therapy in treating genetic disorders, cancer gene therapy, retinal diseases, neuromuscular disorders, hemophilia, and metabolic diseases in humans, as well as inherited and acquired diseases in companion and livestock animals. Despite remarkable progress, challenges related to delivery efficiency, immune responses, off-target effects, manufacturing scalability, regulatory approval, and long-term safety continue to limit widespread clinical implementation. Advances in vector design, genome-editing precision, artificial intelligence-assisted development, and regulatory frameworks are expected to address these limitations. Overall, gene therapy and CRISPRbased technologies represent promising platforms for the development of next-generation precision medicines that could transform disease management in both human and veterinary healthcare.

Abstract from PubMed (PMID 43023515). This entry was added automatically by our daily literature monitor because it matches the topics we follow; read the full paper at the original source.