Genetic-based therapies are discussed in the news nearly every day. These innovative technologies including RNA therapies, gene therapies and gene editing technologies are moving towards the clinical realm at an unexpected pace. They hold the key for 'a cure for all with CF.' However, moving from the promise of these technologies to the reality of effective therapy and cure for all will require time, tenacity and vision. This plenary will take us down that path to review the power of genetic therapies, outline the obstacles/challenges ahead as these therapies move from the pre-clinical space to clinical trials, and discuss what success looks like as we move to a one-time cure.
Review emerging genetic therapies and the promise they hold.
Discuss/Describe the unique challenges we face regarding delivering these therapies in CF.
Discuss/Describe the incredible advantages and resources the CF community will harness to overcome obstacles.