A research team has identified the core gene expression networks regulated by key proteins that fundamentally drive phenomena such as cancer development, metastasis, tissue differentiation from stem ...

Elexacaftor-tezacaftor-ivacaftor (ETI) has significantly improved the clinical course of people with cystic fibrosis ... liver fibrosis indices and liver tests in children with CF aged 6-12 years ...

Importantly, most of the CF gene therapy research is currently funded by charities such as the Cystic Fibrosis Trust, UK; Communication with CF patients and families about progress and ...

Cystic fibrosis is a genetic condition that causes mucus in the body to become thick and sticky. The mucus throughout the body is typically slippery and acts as a protective barrier in the lungs ...

Pfizer gave Sangamo Therapeutics coal for Christmas, terminating a hemophilia A gene therapy pact to deprive the cash-strapped biotech of up to $220 million in milestones. Pfizer’s decision sent ...

Credit: Vertex. According to Vertex Pharmaceuticals, approximately 300 additional people with CF are now eligible to receive treatment. An additional 94 non-F508del CFTR mutations and a boxed ...

Each gene tells a cell to make a specific molecule, but this isn't performed by the DNA itself. Instead, the information is relayed to cellular machinery by a molecule called messenger RNA.

New work by Columbia researchers has turned a textbook principle of genetics on its head and revealed why some people who carry disease-causing genes experience no symptoms. Every biology student ...

"Our study shows that we can deliver genes to renal tubules and podocytes [highly specialized cells] in the kidney, the two important target cell types for gene therapy, and while there is a ...

Pfizer’s research partnership with Sangamo Therapeutics produced a hemophilia A gene therapy that reached FDA discussions about a regulatory submission. That’s as far as the alliance will go.