McConnell Erin M, Holahan Matthew R, DeRosa Maria C
1 Department of Chemistry, Carleton University , Ottawa, Ontario, Canada .
Nucleic Acid Ther. 2014 Dec;24(6):388-404. doi: 10.1089/nat.2014.0492.
Oligonucleotide aptamers are short, synthetic, single-stranded DNA or RNA able to recognize and bind to a multitude of targets ranging from small molecules to cells. Aptamers have emerged as valuable tools for fundamental research, clinical diagnosis, and therapy. Due to their small size, strong target affinity, lack of immunogenicity, and ease of chemical modification, aptamers are an attractive alternative to other molecular recognition elements, such as antibodies. Although it is a challenging environment, the central nervous system and related molecular targets present an exciting potential area for aptamer research. Aptamers hold promise for targeted drug delivery, diagnostics, and therapeutics. Here we review recent advances in aptamer research for neurotransmitter and neurotoxin targets, demyelinating disease and spinal cord injury, cerebrovascular disorders, pathologies related to protein aggregation (Alzheimer's, Parkinson's, and prions), brain cancer (glioblastomas and gliomas), and regulation of receptor function. Challenges and limitations posed by the blood brain barrier are described. Future perspectives for the application of aptamers to the central nervous system are also discussed.
寡核苷酸适配体是短的、合成的单链DNA或RNA，能够识别并结合从小分子到细胞的多种靶标。适配体已成为基础研究、临床诊断和治疗的重要工具。由于其尺寸小、靶标亲和力强、缺乏免疫原性且易于化学修饰，适配体是抗体等其他分子识别元件的有吸引力的替代品。尽管中枢神经系统是一个具有挑战性的环境，但其相关分子靶标为适配体研究提供了一个令人兴奋的潜在领域。适配体在靶向药物递送、诊断和治疗方面具有潜力。在这里，我们综述了适配体在神经递质和神经毒素靶标、脱髓鞘疾病和脊髓损伤、脑血管疾病、与蛋白质聚集相关的病理学（阿尔茨海默病、帕金森病和朊病毒）、脑癌（胶质母细胞瘤和胶质瘤）以及受体功能调节方面的研究进展。描述了血脑屏障带来的挑战和限制。还讨论了适配体应用于中枢神经系统的未来前景。
