Advancing Gene Therapy: Bridging Innovation, Access, and Implementation in Genetic and Rare Diseases

Abstract

This abstract book presents five undergraduate research proposals developed for a genetics research case competition organized by the Global Society for Genetics and Genome Biology Queen’s Chapter at Queen’s University in Kingston, Ontario, Canada. The competition focused on advancing gene therapy by considering scientific innovation, access, implementation, ethics, and equity in genetic and rare diseases. Submissions addressed allogeneic CAR T-cell banking for hematologic malignancies, physician communication training for informed consent in gene therapy, mobile genetics care for Long QT syndrome in the Gitxsan community, telegenetics and patient navigation for sickle cell disease, and an inhalable CFTR mRNA platform for cystic fibrosis. Abstracts were reviewed by the conference planning team, and authors received feedback before final submission. Together, these proposals illustrate undergraduate approaches to translational genetics that consider both therapeutic development and equitable delivery of emerging genomic technologies.