Engineering a versatile lipoMSN delivery platform for the development of gene and drug therapies

The question of delivery has become a critical part of therapeutic research and development. While nanoparticle formulations are now used in a variety of FDA-approved therapies, these focus on simple oral formulations or systemic lipid nanoparticle administration; therefore, significant research is ongoing in the development of specialized delivery systems for more complex cargos or specific targeting[1]. For therapeutics that have high potential for toxicity, like chemotherapeutics, delivery systems need to transport disproportionately into target organs and further target cells. This is more so true for therapeutics that require delivery to a specific cellular compartment to have significant efficacy, like in the case of gene editing in the nucleus or mitochondria-targeted peptides. To contribute to the development of efficient carriers for gene and drug therapy, this work aims to explore the potential of liposome-coated mesoporous silica nanoparticles and engineering methods that provide functionality to defeat barriers to efficient therapeutic development. Recent advances in CRISPR/Cas9 technology present an attractive toolset to study genes for the development of novel therape