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适体靶向寡核苷酸治疗学:一种用于脑递送和治疗神经疾病的更智能方法。

Aptamer-targeted oligonucleotide theranostics: a smarter approach for brain delivery and the treatment of neurological diseases.

机构信息

Nanomedicine-Laboratory of Immunology and Molecular Biomedical Research (NLIMBR), School of Medicine (SoM), Molecular and Medical Research (MMR) Strategic Research Centre, Faculty of Health, Deakin University, Geelong Technology Precinct (GTP), Pigdons Road, Waurn Ponds, Geelong, Victoria 3217. Australia.

出版信息

Curr Top Med Chem. 2015;15(12):1115-24. doi: 10.2174/1568026615666150413153928.

Abstract

Aptamers represent the novel class of oligonucleotides holding multiple applications in the area of biomedicine. The advancements introduced with the Systematic Evolution of Ligands by EXponential enrichment (SELEX) approach further eased the scope of producing modified aptamers within a short span yet retaining the properties of stability and applicability. In the recent times, aptamers were identified to have the potential for penetrating into the deep human crevices and thus can be utilized in addressing the issues of complex neurological disorders. Considering the specificity and stability enhancement by chemical modifications, aptamer-based nanotechnologies may have great potential for future therapeutics and diagnostics (theranostics). The research community has already witnessed success with the approval of macugen (an anti-vascular endothelial growth factor aptamer) for treating degenerating eye disease, and hopefully those that are in the clinical trials will soon be translated for human application. Herein, we have summarized the aptamer chemistry, aptamer-nanoconjugates and their applications against neurological diseases.

摘要

适配体是一类新型的寡核苷酸,在生物医学领域有多种应用。通过指数富集的配体系统进化(SELEX)方法的进步,进一步简化了在短时间内生产修饰适配体的范围,同时保留了稳定性和适用性。最近,适配体被发现具有穿透人类深层缝隙的潜力,因此可用于解决复杂神经疾病的问题。考虑到通过化学修饰提高的特异性和稳定性,基于适配体的纳米技术在未来的治疗和诊断(治疗诊断)方面可能具有巨大的潜力。研究界已经见证了 macugen(一种抗血管内皮生长因子适配体)在治疗退行性眼病方面的成功,希望那些正在临床试验中的药物将很快被转化为人类应用。在此,我们总结了适配体化学、适配体-纳米复合物及其在神经疾病中的应用。

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