Department of Molecular Pharmacology and Experimental Therapeutics, Mayo Clinic College of Medicine and Science, Rochester, Minnesota, USA.
Medical Scientist Training Program, Mayo Clinic Graduate School of Biomedical Sciences and Mayo Clinic Alix School of Medicine, Mayo Clinic College of Medicine and Science, Rochester, Minnesota, USA.
Neuro Oncol. 2024 Jan 5;26(1):38-54. doi: 10.1093/neuonc/noad156.
Despite recent advances in the understanding of brain tumor pathophysiology, challenges associated with tumor location and characteristics have prevented significant improvement in neuro-oncology therapies. Aptamers are short, single-stranded DNA or RNA oligonucleotides that fold into sequence-specific, 3-dimensional shapes that, like protein antibodies, interact with targeted ligands with high affinity and specificity. Aptamer technology has recently been applied to neuro-oncology as a potential approach to innovative therapy. Preclinical research has demonstrated the ability of aptamers to overcome some obstacles that have traditionally rendered neuro-oncology therapies ineffective. Potential aptamer advantages include their small size, ability in some cases to penetrate the blood-brain barrier, inherent lack of immunogenicity, and applicability for discovering novel biomarkers. Herein, we review recent reports of aptamer applications in neuro-oncology including aptamers found by cell- and in vivo- Systematic Evolution of Ligands by Exponential Enrichment approaches, aptamer-targeted therapeutic delivery modalities, and aptamers in diagnostics and imaging. We further identify crucial future directions for the field that will be important to advance aptamer-based drugs or tools to clinical application in neuro-oncology.
尽管近年来对脑肿瘤病理生理学的认识有了进步，但与肿瘤位置和特征相关的挑战阻碍了神经肿瘤学治疗的显著改善。适体是短的、单链 DNA 或 RNA 寡核苷酸，它们折叠成序列特异性的三维形状，与蛋白质抗体类似，与靶向配体具有高亲和力和特异性相互作用。适体技术最近已被应用于神经肿瘤学，作为一种创新治疗的潜在方法。临床前研究已经证明了适体克服一些传统上使神经肿瘤学治疗无效的障碍的能力。潜在的适体优势包括它们的小尺寸、在某些情况下穿透血脑屏障的能力、固有缺乏免疫原性以及适用于发现新型生物标志物。在此，我们回顾了适体在神经肿瘤学中的应用的最新报告，包括通过细胞和体内系统进化的配体的适体，通过指数富集方法，适体靶向治疗传递方式，以及适体在诊断和成像中的应用。我们进一步确定了该领域的关键未来方向，这些方向对于将基于适体的药物或工具推进到神经肿瘤学的临床应用非常重要。
