Disease Focus: Cystic Fibrosis

Overcoming barriers for airway stem cell gene therapy for Cystic Fibrosis

Research Objective This research will allow the targeting of airway stem cells for long lived gene therapy for Cystic Fibrosis and for other airway diseases Impact We will overcome the barriers to accessing airway basal stem cells for gene correction for Cystic Fibrosis (CF) and use a new gene correction strategy to correct >99% of […]

Genome Editing to Correct Cystic Fibrosis Mutations in Airway Stem Cells

Research Objective Gene corrected autologous airway epithelial stem cells from patients with cystic fibrosis to be used as cell and gene based therapy for chronic sinus disease Impact The proposed studies would provide an innovative, readily applied primary stem cell based approach with gene correction to treat chronic sinusitis in CF, a debilitating airway disease. […]