Systematic review, meta-analysis, and future scope of lentivirus-mediated gene therapy

Gene therapy has emerged as a promising therapeutic option for many inherited and acquired diseases. Lentivirus-mediated gene transfer is a powerful technique to deliver therapeutic genes into target cells and offers several advantages over other gene delivery methods. This systematic review explores the mechanistic intricacies of lentivirus-based vectors and provides the current status on their safety and efficacy in therapeutic applications underpinned by meta-analysis of clinical trials reported from across the globe. Our findings elucidate that gene therapy using lentiviruses has been extended to various diseases spanning from immunodeficiencies and hemoglobinopathies to specific malignancies and lysosomal storage diseases. Although the meta-analysis indicates a positive trend with respect to safety, the results pertaining to efficacy were not significant, highlighting the importance of continued investigation and cautious interpretation of the results. The findings from this study underscore the imperative for advancing research in vector development followed by preclinical and clinical assessments, to elevate the potential of lentiviral gene therapy as a potential therapeutic modality in the future.