People with diseases caused by rare mutations have fewer options and poorer prospects than other patients despite rapid ...

Battling a rare mutation of cystic fibrosis, Emily Kramer-Golinkoff fights for her life — and for gene therapies that offer ...

After encapsulating the CRISPR system components including Cas9 mRNA, mutation-specific single guide RNA, and donor single stranded DNA template, they injected the lipid nanoparticles intravenously ...

People living with cystic fibrosis can now take a once-daily drug that will help them breathe easier all day long, thanks to ...

CF is caused by a mutation in the gene for the protein cystic fibrosis transmembrane conductance regulator (CFTR). This gene is required to regulate the components of sweat, digestive juices ...

A new study suggests genetic changes allow horses to produce more energy, while minimizing the toll the energy takes on cells.

States screen for cystic fibrosis (CF) but with significant variation ... the preferred IRT-DNA strategy in the U.S. More than 1,000 mutations are now known to cause CF, but some states test ...

An inhaled gene therapy for cystic fibrosis developed by Boehringer Ingelheim ... One reason for that is that around 2,000 mutations in the CFTR gene can lead to CF, a disease caused by an ...

According to the guidelines, the testing process must be extensive to discover mutations that exist in the Cystic Fibrosis Transmembrane Conductance Regulator(CFTR) gene that creates Cystic ...

With this approval, the indication has been expanded to include all patients ages 2 years and older who have at least one non-class I mutation in the cystic fibrosis conductance regulator (CFTR ...

With this approval, the indication has been expanded to include all patients ages 2 years and older who have at least one non-class I mutation in the cystic fibrosis conductance regulator (CFTR) gene.