Gene therapy using retrovirally transduced human chondrocytes expressing TGF-β1: a systematic review of early results are not promising!

Abstract

Background: Gene therapy is a promising ortho-biologic product for Osteoarthritis (OA) knee with some level 2 and 3 studies. There is a need to evaluate the safety and efficacy of this therapy with a systematic review by pooling the data from the limited clinical trials to give an early idea of the product. Methods: Pubmed, Embase, and Scopus were searched for articles related to gene therapy use in human trials for osteoarthritis using mesh terms. After screening, seven RCTs were included in this review. Clinical outcomes with IKDC scores, VAS, and adverse effects were analyzed. Results: All studies reported significant improvement in functional outcomes measured with IKDC scores. VAS scores showed mixed results. WOMAC scores and WORMS scoring reported no significant difference between the treatment and placebo groups, but the progression of OA was decreased in the treatment group. Conclusion: Gene therapy could potentially improve knee-related functional outcomes and alleviate pain. While some studies indicate positive effects on knee morphology, further research is needed in this regard. Adverse events, though generally manageable, require ongoing vigilance. Gene therapy holds the potential to enhance the quality of life for individuals with osteoarthritis knee.