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 of a cystic ...

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 ...

cystic fibrosis, Alzheimer’s, Duchnene muscular dystrophy (DMD), and other chronic musculoskeletal disorders. The FDA recently approved CRISPR to treat sickle cell disease, an inherited disorder ...

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 ...

Cystic fibrosis (CF) is a life-threatening genetic disease affecting multiple organ systems, with pancreatic dysfunction representing a critical and often overlooked complication.

The bottom line is few companies can match its combination of established leadership in rare diseases and realistic pathways ...