[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 ...

Medically reviewed by Reza Samad, MDCystic fibrosis (CF) treatment typically involves procedures and medications that help ...

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 ...

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 ...

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 ...

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 ...

Together these drugs cover the CFTR mutations seen in around half of all CF patients. The next phase of Vertex is focused on the roll-out of its new triple Kaftrio (tezacaftor, ivacaftor and ...

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 ...