Agenda Details

Learn more about sessions and presentations at the 26th Annual Meeting

Detailed Program

Strategies to Deliver Genetic Therapies for Cystic Fibrosis

Delivery of genetic therapies to the airway epithelium presents unique challenges for a long-lasting therapeutic for people with cystic fibrosis (CF). The CF lung is characterized by thick, sticky mucus that dehydrates due to the loss of functional cystic fibrosis transmembrane conductance regulator (CFTR) channels. Dehydration of the mucus and plugging of the airway lead to reduced lung function and restricted distribution of inhaled aerosolized vector. Systemic delivery likely requires engineered vector tropism and the capacity to navigate from the vasculature to the epithelium. This symposium will bring together leaders in the nanotechnology field to present and discuss novel technologies and strategies to deliver genetic therapies to the lung.

Session Details

Co-Chair

John D. Lueck, PhD. University of Rochester, School of Medicine and Dentistry, Rochester, NY

Co-Chair

Brigitte Gomperts, MD. UCLA, CA

Ferrets as Preclinical Model for Genetic Therapies Development

John F. Engelhardt, Ph.D.. University of Iowa, Iowa City, IA

Cellular Diversity of the Airway

Brigitte Gomperts, MD. UCLA, CA

Nanoparticle-mediated Delivery of mRNA to Airway Epithelial Cells

Gaurav Sahay, PhD. Oregon State University, OR

Polymeric nanoparticle strategies to restore CFTR function

Alexandra S.. Piotrowski-Daspit, Ph.D.. University of Michigan, Ann Arbor, MI

Strategies to Restore Functional CFTR in Epithelial Cells

Sriram Vaidyanathan, PhD. Nationwide Children's Hospital, Columbus, OH

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