Towards gene therapy for cystic fibrosis: enhanced green fluorescent protein as a reporter of promoter activity

Cystic Fibrosis (CF) is the most common lethal inherited disease, affecting -1/2000 live births. Although the genetic lesion, a mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, has been elucidated, the exact mechanism whereby this causes the debilitating disease phenotype is unclear. CF patients are prone to repeated bacterial infection of the lung; leading to fibrosis of this tissue, and eventually respiratory failure. Gene therapy has the potential to cure CF: by introducing a normal copy of the CFTR gene into epithelial cells of the lung, it may be possible to abolish the lung phenotype. While early gene therapy vectors utilised a strong viral promoter (e.g. Pcmv) to drive expression of the CFTR cDNA, it has become apparent that this approach creates problems: expression is short lived and may not be targeted to the correct cell types at appropriate levels.

Grantor dc:publisher

The University of Edinburgh

Year dc:date.issued

2005

Author dc:creator

• Walker, Wendilywn Elizabeth

Handle dc:identifier.uri

http://hdl.handle.net/1842/27597

OAI identifier oai:identifier

oai:era.ed.ac.uk:1842/27597