News

Exon-Skipping Shows Potential to Treat Nonsense Mutations in CF

A team of scientists in New York has created small molecules called antisense oligonucleotides that could be used to “skip over” the mutated part of the CFTR gene to treat cystic fibrosis (CF) patients with a specific “nonsense” mutation and no available therapies. Findings were published in the study, “…

Kaftrio Rescues Chloride, Bicarbonate Deficits in GI Tissues

The triple combination therapy Kaftrio restores the function of CFTR — the defective protein in cystic fibrosis (CF) — and associated chloride and bicarbonate flow in gastrointestinal “mini-organs” derived from a patient carrying the most common CF-causing mutation, a study shows. These findings highlight the benefits of Vertex…

Partial Monocyte Transplant Improves Survival in Mouse Study

Monocyte immune cells from bone marrow partially transplanted into a mouse model of cystic fibrosis (CF) improved survival and reduced inflammation, a study found. Meanwhile, transplanting monocytes from CF mice into healthy mice triggered CF-like symptoms and limited survival, suggesting that these defective immune cells may cause symptoms, the…

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