Related Experiment Video
Updated: Aug 17, 2026

Generation of Human Nasal Epithelial Cell Spheroids for Individualized Cystic Fibrosis Transmembrane Conductance Regulator Study
Published on: April 11, 2018
Pre-clinical and clinical endpoint assays for cystic fibrosis gene therapy
Uta Griesenbach1, A Christopher Boyd,
1Department of Gene Therapy, Faculty of Medicine, Imperial College London, UK. u.griesenbach@imperial.ac.uk
Abstract:
The credibility and hence value of pre-clinical and clinical cystic fibrosis gene therapy studies depend on the assays used to evaluate gene transfer. Awareness of assay suitability, sensitivity and variability is therefore crucial to the design of experimental programmes. Here, we review the assays that are in use to assess the efficacy of gene transfer in pre-clinical and clinical CF gene therapy research, highlight their weaknesses and suggest possible new strategies that may help to overcome current limitations.
More Related Videos
Related Concept Videos
Cystic Fibrosis: Pathogenesis
CF is primarily caused by a genetic mutation in a chromosome 7 gene coding for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. The most common gene mutation leading to CF is the ΔF508 mutation, but...
Gene Therapy
Pharmacogenomics: Identification of New Drug Targets

