Gene editing for cystic fibrosis - tackling the translatability hurdle KU Leuven
This project is focussed on novel gene editing modalities and delivery methods to develop a gene therapy treatment for the monogenetic disease cystic fibrosis which primarily manifests with lung disease. Objectives: (1) To use gene editing (in particular base and prime editing) for efficient correction of CFTR (gene mutations in CFTR are responsible for cystic fibrosis) in primary human organoid and airway models. (2) To develop safe and ...