Sharma J, et al: A novel G542X CFTR rat model of cystic fibrosis is sensitive to nonsense mediated decay ... and to support continued studies of cell-based therapies, gene editing techniques, and ...

Viral and nonviral clinical trials have established proof-of-principle for gene transfer into the cystic fibrosis (CF) lung. It is currently unknown whether gene transfer efficiency is sufficient ...

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 ...

A genetic screen can potentially diagnose more than 1,200 genetic disorders and chromosomal abnormalities. If you were a medical geneticist, how would you pick the best test for your patient?

and we hope future research could lead to the development of genetic therapy treatments for those with rarer CF mutations too,” said Lucy Allen, a lung biologist and research director at Cystic ...

The ACMG charged the Accreditation of Genetic Services Committee, chaired by Dr. Robert Desnick, to establish a Subcommittee on Cystic Fibrosis Carrier ... the screening model to be used (couple ...

An international study has identified two types of these so-called dysbioses in cystic fibrosis. They display ... Landmark Study Involving Babies in Ireland Supports Use of Cystic Fibrosis Drug in ...

An explanation of the cause and effects of the genetic disorder cystic fibrosis and how it can be treated using gene therapy. There is a discussion about gene therapy which includes an interview ...

Vertex Pharmaceuticals (NASDAQ:VRTX), a global biotechnology company with a market capitalization of $119.1 billion known for its transformative cystic fibrosis (CF) treatments, finds itself at a ...

Cystic fibrosis (CF) is a single-gene disorder with insufficient treatment options and a target organ, the lung that is relatively easily accessible. Thus, it is not surprising that in the early ...