An inhaled gene therapy for cystic fibrosis developed by Boehringer Ingelheim is due to start clinical testing shortly, ...

We applied a strategy of serial screening steps to 45 patients with congenital absence of the vas deferens and characterized cystic fibrosis transmembrane conductance regulator gene mutations in ...

Although newborn screening includes CF, this typically only covers a selection of common genetic variants, and with over 2000 reported in the cystic fibrosis transmembrane conductance regulator (CFTR) ...

Until recently, drug development efforts have emphasised therapies treating downstream signs and symptoms resulting from the underlying CF biological defect: reduced function of the CF transmembrane ...

Turning to Sionna, the biotech is focused on the development of novel therapies for cystic fibrosis (CF) that normalise the function of the CFTR protein, whose gene is defective or missing in ...

Pfizer’s decision to halt further marketing of Beqvez is further sign of the sparse patient interest in gene therapies for the bleeding condition. Rapid scientific advances have put the gene therapy ...

Following, the exon upstream of the removed intron is ligated to the 5′ end catalyzed by the spliceosomal RNAs and the ribonuclear proteins (RNPs). Alternative splicing produces variants that differ ...

It seeks to understand the process of trait inheritance from parents to offspring, including the molecular structure and function of genes, gene behaviour in the context of a cell or organism (e.g ...

There are many misconceptions about CF. Learn the facts on our page, Dispelling Misconceptions About Cystic Fibrosis. In people with CF, mutations in the cystic fibrosis transmembrane conductance ...

Copyright 2025 The Associated Press. All Rights Reserved. Chicago Chess Records producer Gene Barge attends a screening of the film “Cadillac Records,” about ...

The management of CF has been changing drastically over the last few years, especially after the introduction of CFTR modulators. Currently, 188,336 people have the disease in 96 countries, but only ...

In this context, endogenous RNAi acts as a natural defence against viral infections and transposable elements (Boudreau et al., 2011). siRNAs are also derived exogenously and are used for gene ...