Gene therapy for cystic fibrosis is advancing fast. Explore key biotech players, clinical progress, and its potential to ...

Single gene disorders, like Huntington’s disease and cystic fibrosis, actually follow Mendelian ... it is much easier to determine how the structure of the corresponding gene product (protein ...

In their 2019 paper, Liu’s team used prime editing to alter the gene mutations causing sickle cell disease ... a lung biologist and research director at Cystic Fibrosis Trust, who was not involved in ...

The disease is caused by mutations in one gene — cystic fibrosis transmembrane conductance regulator, or CFTR — making it, in theory, an ideal candidate for gene-editing technology. However, there’s a ...

The United States Cystic Fibrosis Foundation released the first ... in part because these groups tend to have CF-causing gene variants that are rarer and seldom included in the newborn screening ...

An inhalable medicine with the potential to improve lung disease in people with cystic fibrosis ... known mutations in the CFTR gene, which lead to different levels of the severity of the disease.