The significance of chemical transfection/transduction enhancers in promoting the viral vectors-assisted gene delivery approaches: A focus on potentials for inherited retinal diseases

Viral vectors are among the main approaches currently used in studies for executing gene delivery to target cells. During the past decades of active studies in gene therapy, including viruses, adenoviruses (Ads), lentiviruses (LVs), and adeno-associated viruses (AAVs), have received the most attention among the biological approaches where potentially successful outcomes are recorded for numerous genetic conditions. The success of delivery methods, however, remains unsatisfactory. Using some additives that can improve transgene expression, transfection efficiency, viral particle production, and transduction efficiency is considered as a solution to overcoming the limitations of gene delivery approaches. These additives include caffeine, histone deacetylase (HDAC) inhibitors like sodium butyrate and valproic acid, and polycationic agents like polybrene and protamine sulfate. In this review article, we present an overview of viral vector-mediated retinal gene therapies and the application of some enhancers used to improve the outcomes of gene delivery. Three routes of administrating viral vectors into ocular compartments are employed for the delivery of target genes into impacted cells, and some additives have shown enhanced efficiency of gene delivery in retinal cells. The current study places a special focus on the viral vectors and enhancers used for gene therapies of inherited retinal diseases.

How to cite: Najafi S, Rahimpour A, Ahmadieh H, et al. The significance of chemical transfection/transduction enhancers in promoting the viral vectors-assisted gene delivery approaches: A focus on potentials for inherited retinal diseases. Electron J Biotechnol 2024;72. https://doi.org/10.1016/j.ejbt.2024.07.005.