Optimized microfluidic formulation and organic excipients for improved lipid nanoparticle mediated genome editing
Rohan Palanki, Emily L. Han, Amanda M. Murray, Rohin Maganti, Sophia Tang, Kelsey L. Swingle, Dongyoon Kim, Hannah M. Yamagata, Hannah C. Safford, Kaitlin Mrksich, William H. Peranteau, Michael J. Mitchell
Abstract
relative to the unoptimized LNP formulation. Thus, systematic optimization of LNP formulation parameters revealed a novel LNP formulation that has strong potential for delivery of gene editors to the liver to treat metabolic disease.
Topics & Concepts
MicrofluidicsGenome editingExcipientNanotechnologyIn vivoChemistryComputational biologyMaterials scienceGenomeGeneBiologyBiochemistryChromatographyBiotechnologyCRISPR and Genetic EngineeringVirus-based gene therapy researchRNA Interference and Gene Delivery