Cystic fibrosis is a genetic disorder affecting the lungs and digestive system, causing thick, sticky mucus that can lead to infections. Gene therapy for CF aims to deliver a healthy CFTR gene to ...

People with cystic fibrosis have a gene mutation that causes problems with the protein. Some medications may improve CFTR function. Cystic fibrosis (CF) affects about 70,000 people globally.

In cystic fibrosis (CF), patients’ organs become overwhelmed by thick mucus, which affects breathing and can result in serious bacterial infections. The outlook for patients with CF has improved ...

BETHESDA, Md.--(BUSINESS WIRE)--Today, the Cystic Fibrosis Foundation announced that it will provide up to $15 million to Prime Medicine for preclinical research into gene editing for cystic ...

Though they make up just 1% of cells in the airway, ionocytes could be target cells for gene or cell therapy for cystic fibrosis. Credit: Stuart Rollins, MD, Boston Children’s Hospital ...

This resulted in a high level of gene editing, which boosted levels of the protein made from the cystic fibrosis gene by 5.5-fold, which was enough to restore half of the normal function of the ...

Scientists from Spirovant Sciences describe SP-101, a novel adeno-associated virus (AAV) gene therapy for the treatment of cystic fibrosis (CF) in the peer-reviewed journal Human Gene Therapy.