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适体与 DNA 纳米花结合在神经退行性疾病中的潜在应用。

Potential application of aptamers combined with DNA nanoflowers in neurodegenerative diseases.

机构信息

Beijing Key Laboratory for Bioengineering and Sensing Technology, Daxing Research Institute, School of Chemistry and Biological Engineering, University of Science and Technology Beijing, Beijing 100083, China.

Beijing Key Laboratory for Bioengineering and Sensing Technology, Daxing Research Institute, School of Chemistry and Biological Engineering, University of Science and Technology Beijing, Beijing 100083, China.

出版信息

Ageing Res Rev. 2024 Sep;100:102444. doi: 10.1016/j.arr.2024.102444. Epub 2024 Jul 30.

DOI:10.1016/j.arr.2024.102444
PMID:39084322
Abstract

The efficacy of neurotherapeutic drugs hinges on their ability to traverse the blood-brain barrier and access the brain, which is crucial for treating or alleviating neurodegenerative diseases (NDs). Given the absence of definitive cures for NDs, early diagnosis and intervention become paramount in impeding disease progression. However, conventional therapeutic drugs and existing diagnostic approaches must meet clinical demands. Consequently, there is a pressing need to advance drug delivery systems and early diagnostic methods tailored for NDs. Certain aptamers endowed with specific functionalities find widespread utility in the targeted therapy and diagnosis of NDs. DNA nanoflowers (DNFs), distinctive flower-shaped DNA nanomaterials, are intricately self-assembled through rolling ring amplification (RCA) of circular DNA templates. Notably, imbuing DNFs with diverse functionalities becomes seamlessly achievable by integrating aptamer sequences with specific functions into RCA templates, resulting in a novel nanomaterial, aptamer-bound DNFs (ADNFs) that amalgamates the advantageous features of both components. This article delves into the characteristics and applications of aptamers and DNFs, exploring the potential or application of ADNFs in drug-targeted delivery, direct treatment, early diagnosis, etc. The objective is to offer prospective ideas for the clinical treatment or diagnosis of NDs, thereby contributing to the ongoing efforts in this critical field.

摘要

神经治疗药物的疗效取决于其穿过血脑屏障并进入大脑的能力,这对于治疗或缓解神经退行性疾病(NDs)至关重要。鉴于目前尚无针对 NDs 的明确治疗方法,早期诊断和干预对于阻止疾病进展变得至关重要。然而,传统的治疗药物和现有的诊断方法必须满足临床需求。因此,迫切需要开发针对 NDs 的药物输送系统和早期诊断方法。某些具有特定功能的适体在 NDs 的靶向治疗和诊断中得到了广泛应用。DNA 纳米花(DNFs)是一种独特的花状 DNA 纳米材料,通过环形 DNA 模板的滚环扩增(RCA)复杂地自我组装而成。值得注意的是,通过将具有特定功能的适体序列与 RCA 模板结合,使 DNFs 具有多种功能,从而形成一种新型纳米材料,即适体结合的 DNFs(ADNFs),它融合了两种成分的优势。本文深入探讨了适体和 DNFs 的特性和应用,探讨了 ADNFs 在药物靶向输送、直接治疗、早期诊断等方面的潜在或应用。目的是为 NDs 的临床治疗或诊断提供前瞻性思路,为这一关键领域的持续努力做出贡献。

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