cystic fibrosis

Engineers sharpen gene-editing tools to target cystic fibrosis

by Ian Scheffler, University of Pennsylvania edited by Gaby Clark, reviewed by Robert Egan  Editors’ notes  The GIST Add as preferred source A visualization of the A3G editor’s crystalline structure, with red arrows indicating locations the engineers modified to change their affinity for binding to DNA. Making the editor more accurate could lead to applications like treatments for […]

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Nanoparticle-based gene editing could expand treatment options for cystic fibrosis

by Tiare Dunlap, University of California, Los Angeles edited by Lisa Lock, reviewed by Robert Egan  Editors’ notes  The GIST Add as preferred source Characterization of lipid nanoparticles (LNPs) loaded with CFTR-editing cargoes. Credit: Advanced Functional Materials (2026). DOI: 10.1002/adfm.202502540 UCLA researchers have developed a lipid nanoparticle-based gene-editing approach capable of inserting an entire healthy gene into human airway cells,

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Tiny ‘mini-me’ organs grown from children’s cells are transforming cystic fibrosis care

by University of New South Wales edited by Lisa Lock, reviewed by Robert Egan  Editors’ notes  The GIST Add as preferred source An artistic rendering of airway organoids spiralling around DNA, capturing how each child’s unique biology shapes their path to the right cystic fibrosis treatment. Credit: Shafagh Waters When UNSW Associate Professor Shafagh Waters explains cystic fibrosis

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Study finds cystic fibrosis drug allows patients to safely scale back lung therapies

by University of Colorado Anschutz edited by Sadie Harley, reviewed by Andrew Zinin  Editors’ notes Credit: Pixabay/CC0 Public Domain A new multi-site study led by researchers at CU Anschutz shows that people with cystic fibrosis (CF) who start the triple-drug therapy elexacaftor/tezacaftor/ivacaftor (ETI) can safely reduce many of their daily lung treatments while maintaining good health for years.

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Breaking a sweat: Using chloride in sweat to help diagnose cystic fibrosis

by Ty Tkacik, Pennsylvania State University edited by Gaby Clark, reviewed by Robert Egan The team’s sensor is capable of measuring and reporting the chloride ion levels of a subject’s sweat in real time with high precision and efficiency. Credit: Caleb Craig/Penn State Sweat does more than just cool down an overheating body. Measuring the chemical makeup of an

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Lung damage not main driver of persistent infections in cystic fibrosis, study finds

by University of Washington School of Medicine edited by Sadie Harley, reviewed by Andrew Zinin   Air trapping (colored pink) in lungs of a cystic fibrosis patient before CFTR modulator treatment. Credit: R. Villacreses & D. Stolz Univ. of Iowa A new study meticulously sampled different lung regions in people with cystic fibrosis to understand why infections persist

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Cystic fibrosis: Expert explains symptoms, treatments, causes and more

by Sandy Sufian edited by Sadie Harley, reviewed by Andrew Zinin   Cystic fibrosis (CF) is a rare autosomal recessive disorder that affects numerous systems of the body. It is a complicated disease that differs from person to person. An autosomal recessive disorder is a genetic condition in which an affected person has inherited a mutated gene

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Novel Triple Combination Therapy May Transform Cystic Fibrosis Care in Children

TOPLINE: A 24-week phase 3 trial has found that once-daily vanzacaftor-tezacaftor-deutivacaftor is generally safe and well tolerated in children with cystic fibrosis aged 6-11 years. The treatment maintains normal baseline lung function while improving cystic fibrosis transmembrane conductance regulator (CFTR) function. METHODOLOGY: Researchers conducted a phase 3 trial across 33 clinical sites in eight countries

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