Opportunities, Barriers, and a Strategy for Overcoming Translational Challenges to Therapeutic Nucleic Acid Nanotechnology
ABSTRACT: Recent clinical successes using therapeutic nucleic acids (TNAs) have accelerated the transition of nucleic acid nanotechnology toward therapeutic applications. Significant progress in the development, production, and characterization of nucleic acid nanomaterials and nucleic acid nanoparticles (NANPs), as well as abundant proof-of-concept data, are paving the way toward biomedical applications of these materials. This recent progress has catalyzed the development of new strategies for biosensing, imaging, drug delivery, and immunotherapies with previously unrecognized opportunities and identified some barriers that may impede the broader clinical translation of NANP technologies. A recent workshop sponsored by the Kavli Foundation and the Materials Research Society discussed the future directions and current challenges for the development of therapeutic nucleic acid nanotechnology. Herein, we communicate discussions on the opportunities, barriers, and strategies for realizing the clinical grand challenge of TNA nanotechnology, with a focus on ways to overcome barriers to advance NANPs to the clinic.
摘要：使用治疗性核酸（TNA）的最新临床成功加速了核酸纳米技术向治疗性应用的过渡。核酸纳米材料和核酸纳米颗粒（NANP）的开发，生产和表征方面的重大进展，以及大量的概念验证数据，为这些材料的生物医学应用铺平了道路。这项最新进展推动了生物传感，成像，药物输送和免疫疗法新策略的发展，这些策略以前没有被认识到的机会，并且确定了一些障碍，可能会阻碍更广泛的NANP技术的临床翻译。由Kavli基金会和材料研究协会主办的近期研讨会讨论了治疗性核酸纳米技术发展的未来方向和当前挑战。在这里，我们交流有关实现TNA纳米技术临床挑战的机遇，障碍和策略的讨论，重点是克服将NANP推进临床的障碍的方法。
