Nanotechnologies to Enable Cystic Fibrosis Gene Therapies – Steven Jonas, MD, PhD (Audio)

Dr. Steven Jonas, a pediatric physician scientist and Assistant Professor in the Department of Pediatrics at the UCLA David Geffen School of Medicine and California NanoSystems Institute, presents results from CFRI-funded research on nanotechnologies to enable cystic fibrosis gene therapies. He highlights his team’s approach to overcome common obstacles to gene therapy through the design and testing of CRISPR/Cas9-based gene-editing cargoes for CFTR correction and nanoparticle carriers that can be delivered as aerosolized suspensions to enable inhalable gene therapy solutions for CF. This presentation was part of CFRI's Virtual 35th National CF Education Conference.

CFRI's 35th National CF Education Conference was sponsored by Vertex Pharmaceuticals, Genentech, Gilead Sciences, Chiesi USA, AbbVie, and Ionis Pharmaceuticals.