Modular Assembly of Cell-targeting Devices Based on an Uncommon G-quadruplex Aptamer

Abstract

Aptamers are valuable tools that provide great potential to develop cost-effective diagnostics and therapies in the biomedical field. Here, we report a novel DNA aptamer that folds into an unconventional G-quadruplex structure able to recognize and enter specifically into human Burkitt's lymphoma cells. We further optimized this aptamer to a highly versatile and stable minimized version. The minimized aptamer can be easily equipped with different functionalities like quantum dots, organic dyes, or even a second different aptamer domain yielding a bi-paratopic aptamer. Although the target molecule of the aptamer remains unknown, our microscopy and pharmacological studies revealed that the aptamer hijacks the clathrin-mediated endocytosis pathway for its cellular internalization. We conclude that this novel class of aptamers can be used as a modular tool to specifically deliver different cargoes into malignant cells. This work provides a thorough characterization of the aptamer and we expect that our strategy will pave the path for future therapeutic applications.

DOI: 10.1038/mtna.2015.25

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@inproceedings{Opazo2015ModularAO, title={Modular Assembly of Cell-targeting Devices Based on an Uncommon G-quadruplex Aptamer}, author={Felipe Opazo and Laura Eiden and Line Hansen and Falk Rohrbach and Jesper Wengel and J\orgen Kjems and Guenter Mayer}, booktitle={Molecular therapy. Nucleic acids}, year={2015} }