Science Daily

Prime editing efficiently corrects cystic fibrosis mutation in human lung 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. Now, researchers have developed a ...
Forbes

Internal Nanobodies Tackle Cystic Fibrosis

This voice experience is generated by AI. Learn more. This voice experience is generated by AI. Learn more. A cell-penetrating nanobody restores function in cystic fibrosis—and points to a new class ...
Business Wire

Cystic Fibrosis Rescued by Reprogramming the Cell’s Secretory Machinery

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 ...
Ars Technica

IV infusion enables editing of the cystic fibrosis gene in lung stem cells

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, ...
Phys.org

Nanobody repairs misfolded CFTR inside cells, boosting function in cystic fibrosis

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 ...
Labroots

Efficient base editing in cystic fibrosis airway epithelial cells

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 ...
Medical Xpress

How 'Pac-Man' cells fail to prevent deadly infection risk in people with cystic fibrosis

Researchers have discovered how part of the body's immune system could better combat a leading cause of death for people with cystic fibrosis (CF). A team led by The University of Queensland's ...

Vertex Pharmaceuticals Bumps Up FY Outlook on Growth of Cystic Fibrosis Drugs

The raised outlook comes as Vertex recorded higher sales in its latest quarter, up 12% from a year ago and beating Wall Street's expectations, with the company citing strength for its cystic fibrosis ...
Medical Xpress

Prime editing efficiently corrects cystic fibrosis mutation in human lung cells, study shows

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 ...