Cystic fibrosis (CF) is a life-threatening genetic disease ... The team developed their CF rabbit model using CRISPR/Cas9 ...

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 ...

The Cell Model and Evaluation Core was established in 2007 and is currently organized on the basis of the well-established notion that CF morbidity and mortality can be mitigated by an improved ...

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 ...

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 ...

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

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 ...

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 ...

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 ...