Modulator pills transformed cystic fibrosis care. But the fight for a cure is not over — so long as research remains funded.

After false starts and incremental gains, scientists crack the problem that made breathing impossible. Will answers come fast ...

A systematic review of 52 scientific papers submitted to a world-leading clinical genetics journal from multiple scientists ...

UCLA researchers have developed a lipid nanoparticle-based gene-editing approach capable of inserting an entire healthy gene into human airway cells, restoring key biological function in a laboratory ...

Objectives To determine the diagnostic yield of cystic fibrosis (CF) using a two-tiered genetic testing approach. Although newborn screening includes CF, this typically only covers a selection of ...

Michael Welsh, MD, of University of Iowa Health Care shares how decades of breakthroughs have made normal life possible for many cystic fibrosis patients. Michael J. Welsh, MD, a pulmonologist with ...

Background CFTR modulators have transformed cystic fibrosis (CF) treatment, but individual responses vary even among patients with identical CFTR genotypes. This underscores the need for predictive ...

Cystic fibrosis is a rare genetic disease that affects nearly 40,000 adults and children in the United States. In people with CF, mutations in the CFTR gene result in a buildup of thick mucus in ...

Creative Commons (CC): This is a Creative Commons license. Attribution (BY): Credit must be given to the creator. The Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein, crucial for ...

Advanced biotechnology repurposes two bacterial immune systems to correct large stretches of DNA. Human cells that have been edited with the new retron-based gene editing technology. Orange dots mark ...

While more than 2000 genetic mutations can give rise to cystic fibrosis, the most common is the deltaF508 mutation. 2,8 An estimated 70% to 90% of patients with cystic fibrosis patients have this ...