RT Journal Article
SR Electronic
T1 Gene therapy in cystic fibrosis
JF Archives of Disease in Childhood
JO Arch Dis Child
FD BMJ Publishing Group Ltd and Royal College of Paediatrics and Child Health
SP 465
OP 468
DO 10.1136/archdischild-2012-302158
VO 99
IS 5
A1 Armstrong, David K
A1 Cunningham, Steve
A1 Davies, Jane C
A1 Alton, Eric W FW
YR 2014
UL http://adc.bmj.com/content/99/5/465.abstract
AB The principal cause of morbidity and mortality in cystic fibrosis (CF) is pulmonary disease, so the focus of new treatments in this condition is primarily targeted at the lungs. Since the cloning of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene in 1989, there has been significant interest in the possibility of gene therapy as a treatment for CF. Early studies using viral vectors carrying a healthy CFTR plasmid highlighted the difficulties with overcoming the body's host defences. This article reviews the work on gene therapy in CF to date and describes the ongoing work of the UK CF Gene Therapy Consortium in investigating the potential of gene therapy as a treatment for patients with CF.