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 ...
Science Daily

Prime editing efficiently corrects cystic fibrosis mutation in human lung cells

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. Now, researchers have developed a ...
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 ...
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 ...
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.
Hosted on MSN

Living With A Rare Genetic Mutation: Advanced Cystic Fibrosis

Emily Kramer-Golinkoff, 40, today struggles to take in enough oxygen with each breath. She is living with advances cystic fibrosis, which makes simple activities like walking or showering, exhausting ...
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 ...
American Medical Association

What doctors wish patients knew about cystic fibrosis

Cystic fibrosis treatments are improving patients’ quality of life. Three physicians from University of Iowa Health Care share more about cystic fibrosis.
Healthline

Can Genetic Testing Predict Cystic Fibrosis?

Genetic testing plays an important role in cystic fibrosis screening and diagnosis. It can help determine if a person has cystic fibrosis or carries gene mutations that cause it. Other tests can also ...
Healthline

What You May Need to Know About Cystic Fibrosis Carrier Testing

Genetic testing can help determine if you carry a gene mutation responsible for cystic fibrosis. It may be done using blood, saliva, or cheek tissue samples. The American Lung Association estimates ...
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 ...