Molecular Sciences and Biomedicine Laboratory, State Key Laboratory for Chemo/Biosensing and Chemometrics, College of Chemistry and Chemical Engineering and College of Biology, Collaborative Innovation Center for Molecular Engineering and Theranostics, Hunan University, Changsha 410082, China.
Department of Pharmaceutics and Center for Pharmaceutical Engineering and Sciences, School of Pharmacy, Virginia Commonwealth University, Richmond, VA, 23298, USA.
Theranostics. 2022 Jan 1;12(1):35-47. doi: 10.7150/thno.66466. eCollection 2022.
The past decade has witnessed the blossom of nucleic acid therapeutics and diagnostics (theranostics). Unlike conventional small molecule medicines or protein biologics, nucleic acid theranostics have characteristic features such as the intrinsic ability as "information drugs" to code and execute genetic and theranostic information, ready programmability for nucleic acid engineering, intrinsic stimulatory or regulatory immunomodulation, versatile functionalities, and easy conformational recovery upon thermal or chemical denaturation. Single-stranded circular DNA (circDNA) are a class of single-stranded DNAs (ssDNA) featured with their covalently-closed topology. In addition to the basic advantages of nucleic acids-based materials, such as low cost, biocompatibility, and simplicity of chemical modification, the lack of terminals in circDNA prevents exonuclease degradation, resulting in enhanced biostability relative to the corresponding linear ssDNA. circDNA has been explored for versatile theranostic applications. For instance, circDNA has been extensively studied as templates for bioanalytical signal amplification and the synthesis of nano-/micro-/macro- biomaterials via rolling circle amplification (RCA) and rolling circle transcription (RCT) technologies. circDNA has also been commonly used as the scaffolds for the self-assembly of versatile DNA origami. Finally, circDNA has been implemented as theranostic aptamers, miRNA inhibitors, as well as clustered regularly interspaced short palindromic repeats-CRISPR-associated proteins (CRISPR-Cas) gene editing donors. In this review article, we will discuss the chemistry, characteristic properties, and the theranostic applications of circDNA (excluding double-stranded circular DNA such as plasmids); we will also envision the challenges and opportunities in this research field.
在过去的十年中,核酸治疗和诊断(治疗学)领域取得了蓬勃发展。与传统的小分子药物或蛋白质生物药物不同,核酸治疗学具有内在的作为“信息药物”的特征,能够编码和执行遗传和治疗信息,具有核酸工程的可编程性、固有刺激或调节免疫调节、多功能性以及在热或化学变性后易于构象恢复等特点。单链环状 DNA(circDNA)是一类具有共价闭环拓扑结构的单链 DNA(ssDNA)。除了基于核酸的材料的基本优势,如低成本、生物相容性和简单的化学修饰外,circDNA 缺乏末端,可防止外切核酸酶降解,相对于相应的线性 ssDNA 具有增强的生物稳定性。circDNA 已被探索用于多种治疗应用。例如,circDNA 已被广泛用作生物分析信号放大的模板,并且通过滚环扩增(RCA)和滚环转录(RCT)技术合成纳米/微/宏观生物材料。circDNA 也常用于作为各种 DNA 折纸的自组装支架。最后,circDNA 已被用作治疗学适体、miRNA 抑制剂以及成簇规则间隔短回文重复序列-CRISPR 相关蛋白(CRISPR-Cas)基因编辑供体。在这篇综述文章中,我们将讨论 circDNA(不包括质粒等双链环状 DNA)的化学、特性和治疗应用;我们还将展望该研究领域的挑战和机遇。