Arcturus Therapeutics (NASDAQ:ARCT) is positioning its mRNA platform around two rare-disease programs targeting cystic ...
To generate mice expressing CFTR specifically in ciliated cells, the EGFP cDNA in the previously described FOXJ1 construct 15 was replaced with the cDNA for human CFTR. 17 Transgenic mice were ...
Sionna Therapeutics, Inc. has announced the presentation of promising preclinical data at the 48th European Cystic Fibrosis Conference in Milan, showing that its nucleotide-binding domain 1 (NBD1) ...
A tiny antibody component could fundamentally transform the treatment of cystic fibrosis: For the first time, researchers have succeeded in developing a so-called nanobody that penetrates directly ...
Cystic fibrosis is a hereditary disease caused by a mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The CFTR protein is an ion channel that carries salts into and out ...
A study of 50 adult patients with cystic fibrosis (CF) has found that challenged urine bicarbonate excretion may offer a new, simple, and safe quantification of cystic fibrosis transmembrane ...
The American College of Medical Genetics and Genomics (ACMG) has released updated recommendations for CFTR carrier screening. Pathogenic variants in the CFTR gene can cause cystic fibrosis (CF) as ...
Scientists at St. Jude Children's Research Hospital and Rockefeller University have combined their expertise to gain a better understanding of the cystic fibrosis transmembrane conductance regulator ...
Sionna Therapeutics, Inc. announced that preclinical data on its novel cystic fibrosis (CF) treatment candidates, SION-451 and SION-719, will be presented at the European Cystic Fibrosis Society’s ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results