(166) Iterative High-throughput Screening Identifies Nanoparticles with Improved Delivery Efficiency
Introduction: Neurofibromatosis Type 1 (NF1) is caused by the loss of function of the neurofibromin protein, a tumor suppressor and down-regulator of the RAS signaling pathway. Gene replacement to reconstitute functional neurofibromin presents a promising treatment modality which, due to its wide applicability, has high potential for near-term clinical translation. However, viral vectors do not have the capacity to encapsulate the large NF1 plasmid. To overcome these challenges, we used cationic diblock polymeric nanoparticles (PNPs) to encapsulate and deliver the full length NF1 gene.
Learning Objectives:
Learn about how high throughput screening platforms can be used to identify lead delivery candidates
Ashlee Colbert – Materials Scientist II, Battelle Memorial Institute; Caleb Hillrich – Data Scientist II, Battelle Memorial Institute; Emma Schmitz – Materials Scientist II, Battelle Memorial Institute; Molly Kaufman – Biologist I, Battelle Memorial Institute; Dean Constantine – Chemical Engineer I, Battelle Memorial Institute; Sam Farrar – Chemist I, Battelle Memorial Institute; Andrea McCue – Biologist III, Battelle Memorial Institute; Wallis Deeann – Professor, University of Alabama, Birmingham; Robert Kesterson – Professor, Pennington Biomedical Research Center
