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- Plenary Session I: Thursday, October 31, 2019
"Emerging Technologies for CFTR Restoration in All People With CF"
October 31, 2019
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.
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