Over the next 5 years, a number of outstanding questions will be addressed: The UK CF Gene Therapy ... of-principle for gene transfer into the cystic fibrosis (CF) lung. It is currently unknown ...

Uta Griesenbach, Eric W.F.W. Alton, Department of Gene Therapy, Faculty of Medicine ... proof-of-principle for gene transfer into the cystic fibrosis (CF) lung. It is currently unknown whether ...

Boehringer Ingelheim (BI) and the UK Cystic Fibrosis Gene Therapy Consortium announced a global collaboration to develop a first-in-class, long-term therapy for patients with cystic fibrosis (CF).

Some people with cystic fibrosis still do not have access to the life ... Louise Patterson’s son, Aaron, has a rare gene, which means he is excluded from this therapy. “Currently in Ireland, only gene ...

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 ...

Cystic Fibrosis: A Review. JAMA. 2023;329(21 ... Patients aged 6 years and older who have at least one F508del mutation in the CFTR gene.

Patients aged 6 to 11 years with cystic fibrosis receiving 24-week once-daily vanzacaftor/tezacaftor/deutivacaftor generally ...

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 ...

No single score captures the burden of treatment in people with cystic fibrosis (pwCF), according to systematic review study findings published in the Journal of Cystic Fibrosis.

The implications of mvGPT for gene therapy are significant. The ability to execute multiple genetic edits simultaneously opens new avenues for treating complex conditions. Recent research ...

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 ...

"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 ...