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

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  • Felipe Opazo, Univ Gottingen, University of Gottingen, Med Ctr, Dept Neuro & Sensory Physiol
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  • Laura Eiden, Univ Bonn, University of Bonn, Life & Med Sci Inst
  • ,
  • Line Hansen
  • ,
  • Falk Rohrbach, Univ Bonn, University of Bonn, Life & Med Sci Inst
  • ,
  • Jesper Wengel, Univ Southern Denmark, Nucle Acid Ctr
  • ,
  • Jørgen Kjems
  • Günter Mayer, Univ Bonn, University of Bonn, Life & Med Sci Inst

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.

TidsskriftMolecular Therapy - Nucleic Acids
Antal sider10
StatusUdgivet - 2015

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