Pre-clinical and clinical endpoint assays for cystic fibrosis gene therapy

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.