[1] These mutations are called gating mutations. The CFTR protein reaches the cell membrane but the channel does not open properly and chloride transport cannot occur (Figure 1d). Class IV ...

There are more than 2,000 known mutations in the CFTR gene, which lead to different levels of the severity of the disease. Lentiviral vectors are a type of gene therapy that exploit the ability of ...

Cystic fibrosis (CF) is a life-threatening autosomal recessive disease affecting over 160,000 people worldwide. CF is caused by loss-of-function mutations in the CF transmembrane conductance regulator ...

To understand which cell types had undergone long-lasting editing ... For this, they used a mouse line that carries a mutation in the CFTR gene. Intravenous injections of SORT LNPs carrying a gene ...

UK MHRA approves Vertex’s Alyftrek, a once-daily next-in-class CFTR modulator to treat cystic fibrosis: London Monday, March 10, 2025, 10:00 Hrs [IST] Vertex Pharmaceuticals, a ...

Vertex Pharma receives CHMP positive opinion for label expansion of Kaftrio in combination with ivacaftor: London Tuesday, March 4, 2025, 14:00 Hrs [IST] Vertex Pharmaceuticals, a ...

An inhalable medicine with the potential to improve lung disease in people with cystic fibrosis, irrespective of their mutation type, is being tested in human trials in the UK and Europe. Cystic ...

There are more than 2,000 known mutations in the CFTR gene, which lead to different levels of the severity of the disease. Lentiviral vectors are a type of gene therapy that exploit the ability of ...

There are more than 2,000 known mutations in the CFTR gene, which lead to different levels of the severity of the disease. Lentiviral vectors are a type of gene therapy that exploit the ability of ...