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Published on March 7, 2024
In a bid to create better cystic fibrosis (CF) drugs, Sionna Therapeutics has closed a $182 million Series C financing to develop first-in-class small molecules for this indication. The company’s drugs are designed to fully restore the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein by stabilizing the…
Published on August 29, 2023
Researchers at the University of Michigan have discovered that a rare disease called cystinosis shares the same molecular mechanism as found in a form of cystic fibrosis. This novel finding, reported in the Journal of Clinical Investigation sheds light on how mutated proteins are cleared by the mechanism, impacting the…
Published on June 13, 2023
The American College of Medical Genetics and Genomics (ACMG) has increased the recommended number of pathogenic variants in CFTR gene that can cause cystic fibrosis (CF) from 23 to a set of 100 variants. The new list supersedes the previous smaller group of variants for carrier screening, a form of…
Published on October 26, 2022
Newborn babies with cystic fibrosis who are from non-White ancestry are more likely to be left undiagnosed by healthcare staff, according to research from the University of California. The inherited disorder cystic fibrosis, which causes lung and other organ damage due to excessively thick and sticky mucus, is more common…
Published on October 6, 2022
Gene editing technology that deploys a peptide nucleic acid (PNA) within a biocompatible polymer nanoparticle can correct a common mutation that causes cystic fibrosis (CF), a study in mice has revealed. A single injection partially restored function of the F508del mutation in the CF transmembrane conductance regulator (CFTR) protein, which is…
Published on July 18, 2022
Antisense oligonucleotides, or ASOs, could be used to provide personalized therapy for cystic fibrosis, according to new research by the laboratory of Cold Spring Harbor Professor Adrian Krainer. His team has found how to use ASOs to make more of an imperfect, but still functional, version of CFTR for patients…
Published on February 14, 2022
Research led by the University of Toronto has identified a large group of proteins that interact with the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. The researchers think their findings could clarify why some patients with the inherited condition cystic fibrosis, caused by mutations in the CFTR…
Published on January 12, 2022
In a pair of related PNAS articles, researchers have demonstrated the potential of splice-switching antisense oligonucleotides (ASOs) to eliminate a relatively common CFTR mutation that causes a severe form of the disease. This pair, or cocktail, of ASOs helps the genetic machinery skip over the region encoding the CFTR mutation…
Published on August 9, 2021
An experimental type of gene editing known as ‘prime editing’ can correct mutations that cause cystic fibrosis in human cell lines and organoids in the lab, shows research from the Hubrecht Institute in the Netherlands. While this research is very early stage, it shows that this kind of gene editing…
Published on May 6, 2021
A team of researchers has cataloged healthy lung cells and cells collected from the lung tissue of patients with cystic fibrosis to create a reference database that they hope will give new insights into the inherited condition and help researchers develop new and better treatments. “This new research has provided…
Published on March 19, 2021
Translate Bio said it is continuing its Phase I/II trial of its inhaled mRNA cystic fibrosis (CF) candidate MRT5005 despite it showing a lack of efficacy in a second interim data analysis—a finding that sent the company’s shares tumbling by about one-third yesterday, and led two analysts to express pessimism…
Published on March 22, 2017
University of British Columbia scientists in collaboration with researchers from Drexel University College of Medicine have just published findings of the genomic variation among Burkholderia cenocepacia isolates—one of the most common microbial species found in cystic fibrosis (CF) patients. Chronic lung infections can be devastating for patients with CF and…
Published on July 26, 2016
A recent collaborative study by investigators at Children's Hospital Los Angeles (CHLA), Brigham and Women's Hospital, and the California Department of Public Health has suggested that all infants with a known mutation for cystic fibrosis (CF) and a secondary mutation called the 5T allele, should receive additional screening so as…
Published on July 4, 2016
Imagine yourself suspended a couple of hundred feet below the surface of the ocean. The sheer weight of the immense column of water pushing down on your chest makes each breath a harrowing task. Now picture that your only recourse to collect vital oxygen is to breathe laboriously throw a…
Published on June 9, 2016
A new study led by researchers at Children's Hospital Los Angeles (CHLA) and the Genetic Disease Screening Program of the California Department of Public Health may have shed new light on the 1,800 genetic mutations associated with Cystic fibrosis (CF) that remain uncategorized. CF is a progressive, inherited, autosomal recessive…