A nanobody that stabilizes mutant CFTR from inside the cell, especially when combined with existing drugs, restores near-normal function in cystic fibrosis cells.

Cystic fibrosis is one of the most common genetic disorders, causing thick mucus build-up in the lungs and other parts of the body, breathing problems, and infection. A three-drug cocktail known as ...

The development of gene editing tools, which enable the specific targeting and correction of mutations, hold the promise of allowing us to correct those mutations that cause genetic diseases. However, ...

Researchers have mapped how lung damage begins early in life for children with cystic fibrosis, providing new insights that will help reshape future care. The research team, led by Murdoch Children's ...

BOSTON--(BUSINESS WIRE)--Porosome Therapeutics, Inc. (Porosome Therapeutics) today announced the publishing of a study that brings promise for a cure to cystic fibrosis (CF). The preprint highlights ...

Please provide your email address to receive an email when new articles are posted on . Over a 16-year period, those with cystic fibrosis vs. sickle cell disease had a larger improvement in mean age ...

When UNSW Associate Professor Shafagh Waters explains cystic fibrosis (CF) to the children she works with, she asks them to imagine what is happening inside their own bodies. "I tell them to picture ...

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 ...

A fundamental challenge for cystic fibrosis (CF) gene therapy is ensuring sufficient transduction of airway epithelial cells to achieve therapeutic correction. Gene editing is a method to permanently ...

Australian researchers analysed 190,000 individual cells from 37 kids with cystic fibrosis, building a "lung atlas" of cells in the lower airways of children with the condition. The study found young ...

Despite Vertex Pharmaceuticals' underperformance relative to the healthcare sector over the past year, Wall Street analysts ...

Researchers have mapped how lung damage begins early in life for children with cystic fibrosis, providing new insights that will help reshape future care.