Center for Ultrasound Molecular Imaging and Therapeutics, School of Medicine, University of Pittsburgh, Pittsburgh, PA, 15261, USA.
Department of Pharmaceutical and Administrative Sciences, College of Pharmacy and Health Sciences, Drake University, Des Moines, IA, 50311, USA.
Biotechnol J. 2021 Feb;16(2):e1900408. doi: 10.1002/biot.201900408. Epub 2020 Aug 26.
Nucleic acid-based therapies are promising therapeutics for the treatment of several systemic disorders, and they offer an exciting opportunity to address emerging biological challenges. The scope of nucleic acid-based therapeutics in the treatment of multiple disease states including cancers has been widened by recent progress in Ribonucleic acids (RNA) biology. However, cascades of systemic and intracellular barriers, including rapid degradation, renal clearance, and poor cellular uptake, hinder the clinical effectiveness of nucleic acid-based therapies. These barriers can be circumvented by utilizing advanced smart nanocarriers that efficiently deliver and release the encapsulated nucleic acids into the target tissues. This review describes the current status of clinical trials on nucleic acid-based therapeutics and highlights representative examples that provide an overview on the current and emerging trends in nucleic acid-based therapies. A better understanding of the design of advanced nanocarriers is essential to promote the translation of therapeutic nucleic acids into a clinical reality.
核酸疗法是治疗多种系统性疾病的有前途的疗法,为应对新出现的生物学挑战提供了令人兴奋的机会。最近在核糖核酸 (RNA) 生物学方面的进展拓宽了核酸疗法在治疗多种疾病状态(包括癌症)中的应用范围。然而,包括快速降解、肾脏清除和细胞摄取不良在内的一系列系统性和细胞内障碍,限制了核酸疗法的临床效果。通过利用能够将封装的核酸有效递送至靶组织并释放的先进智能纳米载体,可以克服这些障碍。本文描述了核酸疗法的临床试验现状,并重点介绍了一些代表性实例,概述了核酸疗法的当前和新兴趋势。深入了解先进纳米载体的设计对于推动治疗性核酸转化为临床现实至关重要。