GMO-PMO chimera
Self-transfecting Morpholinos refer to a specialized form of Phosphorodiamidate Morpholino Oligomers (PMOs) that have been engineered to facilitate their own delivery into cells without the need for external transfection agents or delivery systems. This self-transfecting capability simplifies the process of introducing these therapeutic molecules into cells, enhancing their utility for research and therapeutic applications. The modifications that make morpholinos self-transfecting also often enhance their stability and efficacy within cells. This can lead to more effective modulation of target RNA molecules and improved therapeutic outcomes.
Due to their improved cellular delivery, self-transfecting morpholinos are valuable tools in both basic research and clinical applications. They are used to study gene function and have potential therapeutic applications for diseases where efficient and targeted gene silencing is required.