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.

ReCode’s lead programs include RCT2100 for the treatment of the 10% of people with cystic fibrosis who have genetic mutations in the CFTR gene that do not respond to currently approved CFTR ...

New hope for cystic fibrosis patients without treatment options Gomperts has always been driven by a simple goal: finding better treatments for her patients. That pursuit has led her to spend over 20 ...

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 ...

Reference: XJ Wang et al. Mutation in the gene responsible for cystic fibrosis and predisposition to chronic rhinosinusitis in the general population. JAMA 284 (14):1814-1819 (2000).

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.