Development of SORT lipid nanoparticles (LNPs) for genome correction of disease-causing mutations
Tuesday, July 15, 2025
2:29 PM – 2:54 PM EDT
Introduction: In vivo genome correction holds promise for generating durable disease cures. However, to achieve therapeutic editing, controlled delivery vehicles must be developed that can mediate genome correction in specific cells. Moreover, effective stem cell editing remains challenging, necessitating development of delivery nanoparticles that can successfully edit progenitor and stem cells in a tissue and disease relevant context.
Learning Objectives:
At the completion of this activity, participants will know
learn about the origins and genome correction options for genetic diseases.
develop and optimize mRNA lipid nanoparticles (LNPs) for different tissues and applications.
understand nanoparticle targeting mechanisms and broader implications of stem cell editing.
