Carbon Nanotube Applications in Gene Therapy

M. Foldvari
University of Waterloo, CA

Keywords: carbon nanotubes, gene delivery, functionalization

Summary:

Gene therapy, the targeted insertion of DNA coding for a therapeutic gene into the nuclei of diseased cells or tissues followed by its expression, is one of the most promising new therapies for a host of diseases and conditions, yet a number of challenges remain in realizing the full benefit of this treatment mode. In particular, it is necessary to develop gene delivery vectors with targeting ability, high transfection efficiency and improved safety to achieve the full benefit of gene therapy. Pharmaceutical delivery of nucleic acids in vivo is challenging due to the lack of stability, unwanted toxicity and their inability to cross cell membranes. Carbon nanotubes (CNTs) have many potential applications as pharmaceutical excipients. Their unusual properties, in particular their extreme length-to-diameter ratio, propensity for chemical functionalization and potential biocompatibility, make CNTs promising candidates as delivery vehicles. Self-assembling lipid-surfactant-carbon nanotube nanocarriers offer a unique platform where rational design can address the complex constraints of an in vivo environment. Through a systematic modification protocol, coupled with the study of structural and functional relationships between the nano-building blocks, targeting moieties, permeation enhancers (PEs) and plasmid DNA itself, multifunctional biomimetic nanoparticles were developed to reach intracellular target sites.