"Emerging Technologies for CFTR Restoration in All People With CF"
Genetic therapies, including RNA therapies, gene therapies, and gene editing, hold the key to a cure for cystic fibrosis and are moving toward the clinical realm at an unexpected pace. Moving from the promise of these technologies to the reality of effective therapies for the underlying cause of the disease for all people with CF and a cure for CF will require time, tenacity, and vision.
Marie Egan, MD, from the Yale School of Medicine, reviews the power of genetic therapies, outlines the obstacles and challenges that lie ahead, and discusses what success looks like as we move toward therapies that address the underlying defect for all people with CF and a cure for cystic fibrosis.