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

A new inhalable gene therapy for cystic fibrosis (CF) has entered Phase I clinical trials, a major milestone on the journey ...

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 ...

CF null (CFTR-/-) rat: The first CF rat model, CFTRtm1sage, was designed in collaboration ... Electroporation-mediated gene manipulation. To address the role of single gene alterations in airway ...

The patient’s cells must produce the CFTR protein ... fibrosis mouse model harboring the nonsense mutation, G542X. Through next-generation sequencing (NGS) of DNA extracted from its lung tissue, the ...

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 ...

An inhalable medicine with the potential to improve lung disease in people with cystic fibrosis, irrespective of their mutation type, is being tested in human trials in the UK and Europe.

In their 2019 paper, Liu’s team used prime editing to alter the gene mutations causing sickle cell disease and Tay-Sachs disease. Liu explained that they attempted to edit mutated CFTR as well but ...

For example, cystic fibrosis is typically characterized as a single-gene disease associated with recessive mutations in the CFTR gene. However, more extensive studies of CFTR mutations in larger ...

2 CF is caused by mutations in the CFTR gene. These mutations cause the CFTR protein to become dysfunctional, which leads to the development of sticky, thick mucus clogging the airways, which causes ...

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 ...