Medical Xpress

Prime editing efficiently corrects cystic fibrosis mutation in human lung cells, study shows

Cystic fibrosis is one of the most common genetic disorders, causing thick mucus build-up in the lungs and other parts of the body, breathing problems, and infection. A three-drug cocktail known as ...
European Medical Journal

Gene Therapy Breakthrough Restores Key Function in Cystic Fibrosis

UCLA researchers use lipid nanoparticles to insert full CFTR gene into airway cells, restoring function and opening new avenues for CF therapy.
Healio

Q&A: Finding a treatment for rare cystic fibrosis splicing mutation

Please provide your email address to receive an email when new articles are posted on . Few patients with cystic fibrosis have a copy of the 3849+10kb C-to-T splicing mutation. Research is planned to ...
Labroots

A Gene Editor to Correct a Common Cystic Fibrosis Mutation

Cystic fibrosis (CF) is a common genetic disorders that has been well studied. Researchers have identified CF-causing mutations in a gene called CFTR, which encodes for an ion channel. The genetic ...
Yahoo

Genetic medicine can leave people with rare mutations behind. But there's new hope

Add Yahoo as a preferred source to see more of our stories on Google. This photo provided by Emily’s Entourage in April 2025 shows Emily Kramer-Golinkoff, who has cystic fibrosis caused by a rare ...
Technology Networks

Gene Therapy Restores CFTR Function in Cystic Fibrosis Cell Model

Researchers engineered lipid nanoparticles to deliver a full CFTR gene into human airway cells, restoring near-normal ...
News Medical

Yale researchers successfully correct gene mutation that causes cystic fibrosis

Yale researchers successfully corrected the most common mutation in the gene that causes cystic fibrosis, a lethal genetic disorder. The study was published April 27 in Nature Communications. Cystic ...
Technology Networks

Improved Gene Editing Tool Reduces Off-Target Mutations by 80%

A refined base-pair editor limits unintended neighboring DNA edits while maintaining strong correction of disease-causing ...
Monthly Prescribing Reference

Inhalation Therapy Fast Tracked for Cystic Fibrosis With a Specific Mutation

SPL84 is administered directly into the lungs where it is taken up by the cells and is expected to drive the production of fully functional CFTR proteins. The Food and Drug Administration (FDA) has ...
Healio

Cystic fibrosis triple therapy improves clinical outcomes in N1303K mutation

Please provide your email address to receive an email when new articles are posted on . Sweat chloride did not significantly differ between baseline and day 28 of triple therapy in this patient ...
News 13

Genetic medicine can leave people with rare mutations behind, but there's new hope

NATIONWIDE — Emily Kramer-Golinkoff can't get enough oxygen with each breath. Advanced cystic fibrosis makes even simple things like walking or showering arduous and exhausting. She has the most ...
Newsday

Genetic medicine can leave people with rare mutations behind. But there's new hope

Emily Kramer-Golinkoff can’t get enough oxygen with each breath. Advanced cystic fibrosis makes even simple things like walking or showering arduous and exhausting. She has the most common fatal ...