Introduction: Nucleic acid-based therapies are promising advancements in medicine. They offer unparalleled efficacy in treating previously untreatable diseases through precise gene manipulation techniques. However, the challenge of achieving targeted delivery to specific cells remains a significant obstacle.
Areas covered: This review thoroughly examines the physicochemical properties of nucleic acids, focusing on their interaction with carriers and exploring various delivery routes, including oral, pulmonary, ocular, and dermal routes. It also examines the nonviral vector delivery efficiency of nucleic acids, focusing on RNA, and provides regulatory landscapes.
Expert opinion: The role of carriers in improving the effectiveness of nucleic acid-based therapies is emphasized. The discussion of published results covers regulatory frameworks, including insights into European Medicines Agency guidelines. It highlights cutting-edge biotechnological innovations and a quality-by-design approach that could facilitate clinical translation and smooth regulatory obstacles.
Keywords: Delivery vector; gene therapy; nanotechnology; nonparenteral administration; quality-by-design; regulatory requirements.