The first CF rat model, CFTR tm1sage (termed CFTR-/- or KO), was designed in collaboration with SAGE ... and to support continued studies of cell-based therapies, gene editing techniques, and ...

This model avoids the cost of specimen collection ... Given what we now know about the CFTR gene and its phenotypic effects, it is certain that many complicated laboratory and counseling ...

The CFTR gene was identified in 1989, but few companies jumped at the chance to use the information to develop new therapies, because the market was perceived as too small. It is therefore ...

CF null (CFTR-/-) rat: The first CF rat model, CFTRtm1sage, was designed in collaboration with SAGE (now Envigo ... and is highly diminished in CF animals. Electroporation-mediated gene manipulation.

This model offers an unprecedented opportunity ... CF is caused by mutations in the CF transmembrane conductance regulator (CFTR) gene, resulting in abnormal chloride and sodium transport across ...

The patient’s cells must produce the CFTR protein for the existing therapies ... intravenously into a cystic fibrosis mouse model harboring the nonsense mutation, G542X. Through next-generation ...

A new inhalable medicine, potentially improving lung disease in all cystic fibrosis patients, is in human trials across the UK and Europe (1 Trusted Source A Study to Test How Well BI ...