RNA Editing Laboratory, Oncohaematology Department, IRCCS Ospedale Pediatrico Bambino Gesù (OPBG), 00146 Rome, Italy.
Institute of Translation Pharmacology, National Research Council of Italy (CNR), 00133 Rome, Italy.
Molecules. 2020 Sep 17;25(18):4267. doi: 10.3390/molecules25184267.
Glioblastoma (GBM) is the most aggressive, infiltrative, and lethal brain tumor in humans. Despite the extensive advancement in the knowledge about tumor progression and treatment over the last few years, the prognosis of GBM is still very poor due to the difficulty of targeting drugs or anticancer molecules to GBM cells. The major challenge in improving GBM treatment implicates the development of a targeted drug delivery system, capable of crossing the blood-brain barrier (BBB) and specifically targeting GBM cells. Aptamers possess many characteristics that make them ideal novel therapeutic agents for the treatment of GBM. They are short single-stranded nucleic acids (RNA or ssDNA) able to bind to a molecular target with high affinity and specificity. Several GBM-targeting aptamers have been developed for imaging, tumor cell isolation from biopsies, and drug/anticancer molecule delivery to the tumor cells. Due to their properties (low immunogenicity, long stability, and toxicity), a large number of aptamers have been selected against GBM biomarkers and tested in GBM cell lines, while only a few of them have also been tested in in vivo models of GBM. Herein, we specifically focus on aptamers tested in GBM in vivo models that can be considered as new diagnostic and/or therapeutic tools for GBM patients' treatment.
胶质母细胞瘤（GBM）是人类最具侵袭性、浸润性和致命性的脑肿瘤。尽管近年来在肿瘤进展和治疗方面的知识有了广泛的进步，但由于难以将药物或抗癌分子靶向到 GBM 细胞，GBM 的预后仍然非常差。改善 GBM 治疗的主要挑战涉及开发一种靶向药物递送系统，该系统能够穿过血脑屏障（BBB）并特异性靶向 GBM 细胞。适体具有许多使其成为治疗 GBM 的理想新型治疗剂的特征。它们是短的单链核酸（RNA 或 ssDNA），能够与高亲和力和特异性结合分子靶标。已经开发了几种针对 GBM 的适体用于成像、从活检中分离肿瘤细胞以及将药物/抗癌分子递送到肿瘤细胞。由于其特性（低免疫原性、长稳定性和低毒性），已经针对 GBM 生物标志物选择了大量适体，并在 GBM 细胞系中进行了测试，而只有少数适体也在 GBM 体内模型中进行了测试。在这里，我们专门关注在 GBM 体内模型中进行测试的适体，这些适体可以被视为 GBM 患者治疗的新的诊断和/或治疗工具。
