PTAC/CFTR
Alexandra Piotrowski-Daspit, Ph.D.
University of Michigan
Stephen Hart
UCL Great Ormond Street Institute of Child Health
Department of Genetics and Genomic Medicine, UCL Great Ormond Street Institute of Child Health,London, UK
The development of effective genetic therapies for cystic fibrosis (CF) hinges on successful delivery to airway epithelial cells, the principal sites of lung pathology. This session delves into nonviral systems that aim to overcome the specific challenges associated with targeting therapeutic delivery to these cells. Attendees will gain insights into the latest advancements in the design and optimization of nonviral vectors for CF therapeutics, including the selection of materials (ex. lipids or polymers) and targeting ligands to enhance cellular uptake and transfection. The session will also cover strategies to address physiological barriers and promote sustained therapeutic benefits. Overall, this session will highlight the promise of nonviral approaches, address current limitations, and outline the potential directions for translating these strategies into clinical applications for CF.