UAB Lab Experience
My husband & I went to Birmingham in 2024 to tour the UAB Research Facility where groundbreaking research is being conducted on Cystic Fibrosis. We were able to meet with the scientists performing this research & actually walk around the lab where these experiments are taking place. We were able to see three in particular.
The first one looked at the lung cells & how the cilia of the lung cells move mucus in people with CF vs nonCF. (Cilia is like a conveyor belt, moving things along. The stickier mucus makes it hard for the cilia to move it out, allowing it to get stuck.).
The second one is studying mucus & how it is changing with the help of the new modulators like Trikafta to thin the mucus.
The 3rd experiment we saw was on gene splicing & how it changes CF lung cells ability to function more normally. Gene splicing is inserting the correct gene in the place of the defected CF one. One thing that stood out to us was that this particular lab had developed a way to take any cell in the body & turn it into a stem cell that can then be converted into lung cells to advance research. This alleviates the ethical debate with using embryonic stem cells.
Finally, while we did not tour this part of the lab, they think within 5 years they will begin human trials involving gene editing with the goal of correcting the genetic defect in the CFTR gene. This may effectively cure rather than treat Cystic Fibrosis. This is similar to the recent development of a genetic cure of Sickle Cell. This research is amazing & so hopeful for the future of CF & other genetic diseases. It’s awesome to actually see where donations to the Cystic Fibrosis Foundation are making a difference. CFTR gene -chloride transporter that controls salt, which controls water. This does not work correctly which allows mucus to get sticky & build up. Also causes them to get dehydrated easier than non-CF people.
Aside from the lab tour, we were also able to meet other CFF staff & CF parents who understand the journey & struggles of life with this disease. CF sucks & I wish Emmett’s life was different but through this journey we are meeting some incredible people. We are so thankful for this opportunity & would encourage anyone who wants to learn more to go.