Peptide-Lipid Nanoparticle Platform for Cas9-Mediated Gene Editing

Abstract

This thesis explores cell penetrating peptides as delivery vehicles for gene therapy. The primary objective of this work is to characterize the impact of PepFect14 saturated fatty acid modifications on the peptide’s ability to deliver CRISPR/Cas9 ribonucleoprotein into cells and mediate gene editing. Through physicochemical characterization, membrane activity, cell viability and gene editing efficiency studies this thesis identifies the length of saturated fatty acid modification of PepFect14 as an important parameter in its efficiency to facilitate gene editing. It was found that longer saturated fatty acid tail is beneficial for stable nanoparticle formation, cargo encapsulation and gene editing efficiency.