Highly compacted pH-responsive DNA nanoparticles mediate efficient transgene silencing in experimental glioma

Anthony J. Kim1,2, Nicholas J. Boylan2, Jung Soo Suk2, Justin Hanes2, and Graeme F. Woodworth1,2

1Department of Neurosurgery, University of Maryland School of Medicine, Baltimore, MD, 21201, USA; 2The Center for Nanomedicine, Johns Hopkins School of Medicine, Baltimore, MD, 21231, USA

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Summary
Complex genetic mutations are common in brain cancer, making gene therapy an attractive approach to repair or modulate altered genes and cellular pathways. We engineered a pH-responsive DNA nanoparticle platform designed to protect DNA from degradation in vivo, enable efficient intracellular trafficking and transgene expression, and tested the ability of this system to knockdown a model gene in brain tumors.

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