Patricia Inácio, PhD, science writer —

In a groundbreaking study, scientists reported editing and correcting, for the first time, a defective gene in human embryos. Additionally, this gene-editing technique appeared to be safely performed without introducing additional harmful genetic alterations. These results represent a key achievement in human genetic engineering and highlight a potential way…

A substance found in a Japanese cypress tree can deliver iron to cells that are unable to obtain it because of faulty proteins, a finding that has implications for cystic fibrosis, according to a study. Researchers said their molecular-prosthetics approach could not only address cystic fibrosis (CF), but also other conditions…

Researchers at TGV-inhalonix said their company’s drug, Mul-1867, has a powerful effect against clinical isolates of fungi in patients with cystic fibrosis (CF) and other severe lung infections. The study “In vitro activity of a novel compound, Mul-1867, against clinically significant fungi Candida spp. and Aspergillus spp.” appeared in the International Journal of Antimicrobial…

Scientists at Boston University’s Center for Regenerative Medicine have made two stem cell discoveries that could help doctors treat cystic fibrosis (CF) without having to resort to transplants. They prevented stem cells from creating cells for a lot of organs at once and producing only the lung cells they need…

Vertex Pharmaceuticals recently announced positive results of two Phase 3 clinical trials, the EVOLVE and EXPAND studies, evaluating the effects of a combination of tezacaftor (VX-661) and ivacaftor (sold under the brand name Kalydeco) in cystic fibrosis (CF). The results showed that the new combined therapy significantly improved…

Kalydeco (ivacaftor, VX-770) improves bicarbonate secretion in cystic fibrosis (CF) patients with a certain gene mutation, according to a study. Bicarbonate is essential to digestion and the flow of several secretions in the body, including those of the intestine and pancreas. The report, “Impact of CFTR…

Scientists at the University of California-Riverside have developed a novel way to help target therapeutics for cystic fibrosis (CF) and other diseases. Their study, “Inhibition of nonsense-mediated RNA decay by ER stress,” appeared in the journal RNA. The information encoded in human DNA is first converted to RNA, and…

Rare mutations in the CFTR gene — which is defective in patients with cystic fibrosis (CF) — may occur in specific ethnic groups, suggests a new study that urges wider screening of mutations in patients who test negative for most previously identified mutations linked to CF. The study, “A rare CFTR mutation…

A mouse model that reproduces the human symptoms of cystic fibrosis (CF) and chronic obstructive pulmonary disease (COPD) gives scientists a new way to study, and perhaps develop treatments for, the diseases, according to new research. Researchers at Kumamoto University in Japan developed the model, then tested it to see how…

Although patients with cystic fibrosis (CF) often have low levels of the antioxidant glutathione (GSH), the clinical benefits of GSH as an inhaled therapy appear to depend on the inflammatory status of their airways. The study, “Increasing sputum levels of gamma-glutamyltransferase may identify cystic fibrosis patients who do not…