Nucleic acid-based therapies represent a transformative modality for medical treatments. Early clinical success, demonstrated by the approval of several nucleic acid drugs, has been achieved through persistent technological innovation and effective adherence to two pivotal principles: (i) understanding the molecular mechanisms of nucleic acid action and cellular transport, and (ii) engineering effective delivery systems with desired tissue or cell specificity. Advances in this area have made nucleic acid therapeutics for liver diseases a reality. This Keystone Symposia conference on Nucleic Acid Therapeutics and Targeted Delivery focuses on the frontiers in this field, across several areas: advancements in chemistry and targeted delivery, exploration of novel mechanisms of therapeutic gene regulation, and the integration of informatics and AI-based design strategies to optimize systems for success. The diversity of nucleic acid molecular mechanisms—ranging from semi-permanent gene regulation to readthrough codon skipping and gene editing modalities—enhances possibilities for applications and innovative treatments. Decades of research have demonstrated how nucleic acid modification chemistries, fundamental delivery principles, and intracellular interactions provide guiding principles to understand and optimize RNA-based drugs. To harness the translational potential of these emerging mechanisms, the current conference will attract a diverse audience comprising experts in chemistry, cellular and molecular biology and pharmacology to foster interdisciplinary exchange and cross-pollination of ideas. This conference will be held jointly with the Keystone Symposia conference on Precision Genome Engineering: From Basic Mechanisms to Application, which is intended to focus on the intersections of mechanisms and technologies that will enable the selective delivery of nucleic acids to therapeutically relevant cell types and explore the emerging challenges and solutions to extend the success of genetic drugs.  Through shared keynote and joint scientific sessions, meals and poster sessions, participants will have an opportunity to network and share cross-disciplinary insights, encouraging innovative collaborations towards enhancing the development and delivery of nucleic acid-based drugs. 

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