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

Category
Nanoparticle Based Delivery to the Brain
Year
2014
Authors
Anthony J. Kim Nicholas J. Boylan, Jung Soo Suk, Justin Hanes, and Graeme F. Woodworth
Institutions
Department of Neurosurgery, University of Maryland School of Medicine, Baltimore, MD, 21201, USA; The Center for Nanomedicine, Johns Hopkins School of Medicine, Baltimore, MD, 21231, USA
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.