A randomised, double-blind, placebo-controlled phase IIB clinical trial of repeated application of gene therapy in patients with cystic fibrosis
Alton EWFW., Boyd AC., Cheng SH., Cunningham S., Davies JC., Gill DR., Griesenbach U., Higgins T., Hyde SC., Innes JA., Murray GD., Porteous DJ.
The UK Cystic Fibrosis Gene Therapy Consortium has been working towards clinical gene therapy for patients with cystic fibrosis for several years. We have recently embarked on a large, multi-dose clinical trial of a nonviral, liposome-based formulation powered for the first time to detect clinical benefit. The article describes the details of the protocol.