Rational Nanoparticle Design for Efficient Transmucosal Gene Delivery

Cystic fibrosis (CF) is the most common fatal genetic disease affecting Canadian children and young adults, without a cure. Nanoparticles (NPs) have emerged as a promising strategy for delivering genetic treatments for various diseases. However, the development of NPs to treat CF has encountered many obstacles. This is mostly because the human airways, which constantly secrete mucous, pose a challenging environment for the efficient NP-mediated delivery of gene therapy. Several strategies have been proposed and tested; however, rational design criteria for these NPs are still missing. Hence, the researchers, working closely with Providence Health Care, propose a two-pronged interdisciplinary approach (both modeling/simulations and experimental validation) to determine the properties required of the NPs for successful delivery in healthy and diseased tissue models, as well as to develop the criteria for rational NP design. We hope that this work will lead to a breakthrough in NP treatments for CF and other genetic diseases.

Faculty Supervisor:

Sarah Hedtrich;James J Feng


Mohammad Reza Rokhforouz


Providence Health Care


Engineering - chemical / biological



University of British Columbia


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