RESUMEN
DNA vector-based short hairpin RNA (shRNA) as a means of effecting RNA interference (RNAi) is a promising mechanism for the precise disruption of gene expression to achieve a therapeutic effect. The clinical usage of shRNA therapeutics in cancer is limited by obstacles related to effective delivery into the nuclei of target cancer cells. Significant pre-clinical data have been amassed about biodegradable and non-biodegradable polymeric delivery vehicles that are relevant for shRNA delivery into humans. Here, we will review some leading candidates for clinical usage with a focus on studies relating to their potential for usage in cancer shRNA therapeutics and discuss some of the advantages and disadvantages of using biodegradable and non-biodegradable delivery vehicles.
Asunto(s)
Neoplasias/terapia , Polímeros/metabolismo , ARN/uso terapéutico , Silenciador del Gen , Humanos , Interferencia de ARNRESUMEN
The use of DNA vector-based short hairpin (sh)RNA for RNA interference shows promise as a precise means for the disruption of gene expression to achieve a therapeutic effect. The in vivo usage of shRNA therapeutics in cancer is limited by obstacles related to effective delivery into the nuclei of target cancer cells. Nonviral delivery vehicles that are relevant for shRNA delivery into humans belong to a group of substances about which significant preclinical data has been amassed to show an acceptable safety profile, resistance to immune defenses and good transfection efficiency. Here, we review the most promising current nonviral gene delivery vehicles with a focus on their potential use in cancer shRNA therapeutics.