People with cystic fibrosis have better treatment options ... but the Foundation is also enthusiastic about technologies like CRISPR Cas9 to selectively correct mutated genome sequences.

Insufficient CFTR levels affect ion transport in the lungs and contribute to the development of cystic fibrosis. The researchers programmed a type III CRISPR to target the mutant CFTR transcripts. The ...

View Full Profile. Learn about our Editorial Policies. In a study published in Nature Communications, scientists reported a novel strategy to deliver the CRISPR-Cas9 gene-editing system into the lungs ...

Vertex continues to capitalize on its leadership in cystic fibrosis (CF) treatments, where its blockbuster drug Trikafta is ...

“Think about it like breathing in a CRISPR-Cas9 gene-editing package,” said Jonas, an assistant professor of pediatrics at UCLA. UCLA researchers are developing an innovative gene-editing platform ...

Cystic fibrosis is one of the most prevalent genetic ... which is a variation of the genetic snipping tool Crispr. The new ...

For example, in the treatment of monogenic diseases like cystic fibrosis, sickle cell disease, and osteogenesis imperfecta, CRISPR technologies have shown promise in correcting disease-causing ...

The programme utilises Intellia's clustered regularly interspaced short palindromic repeats (CRISPR)-based gene ... "ReCode to secure funds for cystic fibrosis gene correction treatments" was ...

CRISPR Therapeutics already has financial backing from Celgene and in October announced a discovery alliance with Vertex in cystic fibrosis. Novartis has a stake in rivals Intellia Therapeutics ...

Vertex forecasts upbeat 2025 revenue on cystic fibrosis treatments demand Vertex ... 2024 UK's NHS to roll out Vertex/CRISPR gene therapy for rare blood disorder Britain's National Health Service ...