Modern Approaches in GeneTherapy: Mechanisms, Vectors, andTherapeutic Perspectives

Abstract

Gene therapy is the new approach to revolutionize the latest medical practice, to treat and prevent diseases by repairing or adjusting the malfunctioning genes on the molecular level. Gene therapy treats the cause of diseases, unlike conventional therapies, which mainly relieve the symptoms of the diseases, which presents an opportunity of long-lasting and therapeutic effects of the diseases. Gene delivery systems are quite effective and safe in regard to the efficacy of gene therapy. A wide range of viral vectors, including adenovirus, adeno-associated virus, lentivirus, retrovirus and herpes simplex virus, and non-viral vectors, including plasmid DNA, liposomes, polymeric nanoparticles and exosomes have been explored to increase the efficiency of gene transfer and reduce the toxicity and immunogenicity also, there are physical methods including electroporation, sonoporation, magnetofection, optoporation, gene gun and microinjection, which have enhanced localized gene delivery and therapeutic accuracy. Improvements in using gene therapy have been witnessed in cardiovascular diseases, cancer, and neurological disorders. Gene therapy stimulates angiogenesis and myocardial repair in cardiovascular diseases. Suicide gene therapy, microRNA-based therapy, nucleic acid therapy and oncolytic virus therapy are some of the approaches that have shown promise of tumor-targeting ability in cancer treatment. Gene therapy provides a possible cure in neurological diseases to counter the barrier of blood-brain and the low rate of neuronal regeneration. The sustained innovations in the development and targeting of vectors are widening the clinical potential of gene therapy and making it a promising therapeutic platform in the treatment of complex and hitherto untreatable diseases.