VOL. XXXIX | SPRING 2026
Cystic Fibrosis Center
NEWS & NOTES
NEWS FROM THE FRONT
Inside this issue… News from the Front What's News Patient Spotlight Parent to Parent Clinician's Corner Team Updates Research Updates
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Future Cystic Fibrosis Treatment with New Science: mRNA and CRISPR Explained Samya Z. Nasr, MD, CF Center Director
What Is Cystic Fibrosis (CF)? Cystic fibrosis is a genetic disease, meaning it’s caused by changes in a person’s DNA resulting in CF. This change keeps the body from making a healthy protein called Cystic Fibrosis Transmembrane Conductance Regulator (CFTR). Without healthy CFTR, mucus gets thick and sticky, which causes breathing problems, infections, and trouble absorbing nutrients. Since the diagnosis of CF, doctors managed CF with medications, physiotherapy, and special diets, but couldn’t fix the actual problem—the faulty gene. Now, two exciting new types of treatment—mRNA (messenger RiboNucleic Acid) -based therapy and CRISPR (Clustered Regulatory Interspaced Short Palindromic Repeat) gene editing—are being developed to target the disease at its source!
That would especially help treat people with CF (pwCF) who don’t qualify for the CFTR modulators, like pwCF who have class 1, and others who develop side effects from the modulators or are not benefiting from them.
What’s So Special About This?
mRNA-Based Therapies: Teaching Cells to Make the Right Protein
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Imagine cells as factories. In a healthy person, each cell follows a recipe (your DNA) to make the correct CFTR protein. In CF, the recipe has a typo, so the factory turns out a broken protein.
What’s Happening Now?
mRNA therapy hands the cell a fresh copy of the correct recipe.
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How Does It Work? •
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Scientists make healthy CFTR instructions using mRNA (think of mRNA as a temporary blueprint). They package these instructions into tiny bubbles (called lipid nanoparticles) or other ways to carry the healthy CFTR into the lungs, which keep them safe in the body. CF patients can breathe in these bubbles as a mist, letting them reach lung cells. The lung cells read the new mRNA instructions and start making the healthy CFTR protein—just like a factory following a corrected recipe.
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It doesn’t change your DNA, so it’s safe and temporary. It could help anyone with CF, no matter what specific genetic typo they have. The effect wears off after a while, so treatments need to be repeated.
Clinical trials and studies show it’s safe, and some patients start making more healthy CFTR protein in their airways. Scientists are working to make mRNA last longer, work better, and be easier to give.
CRISPR Gene Editing: Fixing the Recipe Forever Now, imagine you could walk into the factory and fix the error in the actual recipe, so the cell makes the right protein from now on. That’s what CRISPR gene editing aims to do.
How Does CRISPR Work? • • •
CRISPR is like GPS-guided scissors for your DNA. It finds the spot where the CF gene has a typo. It snips the DNA at the mistake, then helps the cell patch it up with the correct sequence—or even insert healthy genetic material.
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