news
Prime editing corrects the CFTR gene mutation
The approach precisely and durably corrects the CFTR mutation in human lung cells, which could lead to superior treatments.
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Cystic fibrosis (CF)
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New ENaC blocker demonstrates an extended duration of action
The novel drug, ETD001, could provide an improved approach for mucus clearance in cystic fibrosis patients.
news
Engineered biofilm helps to tackle infections in CF patients
A living material resembling sputum from CF patients can grow biofilms, enabling scientists to assess the effectiveness of antimicrobials.
news
Pulmonary ionocytes: unexpected discovery has implications for CF
Researchers found surprising function of pulmonary ionocytes which may affect how cystic fibrosis drugs work.
article
Right model, right complexity: using in vitro human disease cell models in drug discovery
Ensuring that drug candidates can reach the clinic is no easy task, so having models that can closely represent human pathology is crucial. Here, Dr Beth Hoffman, CEO of Origami Therapeutics, describes the successes and challenges of using human disease cell models in drug discovery.
news
Prime editing method corrects cystic fibrosis in stem cells
A team of scientists used a CRISPR-Cas9 technique known as prime editing to correct cystic fibrosis in cultured human stem cells.
article
New models for intranasal drug delivery
In this article, Dr Jon Volmer and Dr Jon Lenn discuss a new approach to formulation models to test drug delivery based on reconstituted nasal epithelium.