Improving the efficacy of lung delivery of gene editors through optimized lipid nanoparticle formulations to repair cystic fibrosis-associated mutations in human lungs

Cystic fibrosis (CF) is one the most common life-shortening inherited diseases. In the past, patients rarely survived into adulthood; and currently, even with treatment, patients still often require difficult lung transplants. New drugs called protein modulators are a game-changer for CF patients; however, they require twice daily lifelong administration and cannot offset the pre-existing lung damage. Importantly, these drugs are also not effective for all patients, leaving a significant gap in the treatment options. Researchers have recently begun to demonstrate the utility of new gene editing technologies to correct CF-causing mutations. However, critical gaps remain in demonstrating therapeutic levels of correction in patient lung cells, in using clinically relevant delivery strategies, and in establishing the safety and specificity of gene editing technologies. This project aims to use patient lung cells, the gold-standard predictive model in CF drug development, to evaluate and compare new gene editing and delivery technologies. Broadly, this research will aid the development of future gene editing strategies for lung diseases, and specifically will provide the pre-clinical validation for new gene editing treatments for CF.

Faculty Supervisor:

Colin Ross;Sarah Hedtrich

Student:

Partner:

Acuitas Therapeutics

Discipline:

Life Sciences

Sector:

Professional, scientific and technical services

University:

The University of British Columbia

Program: