Gene therapy progress and prospects: cystic fibrosis.

Griesenbach U, Ferrari S, Geddes DM, Alton EW

Gene Therapy

Gene Ther. 2002 Oct;9(20):1344-50.

 Download the Manuscript

Contact us about this publication

Print this page

Since the cloning of the cystic fibrosis gene (CFTR) in 1989, 18 clinical trials have been carried out, including five in the 2 years reviewed here. Most trials demonstrated proof-of-principle for gene transfer to the airway. However, gene transfer efficiency with each of the three gene transfer agents (adenovirus (Ad), adeno-associated virus 2 (AAV2) and cationic liposomes) was low, and most likely insufficient to achieve clinical benefit. Here, we will review the clinical and pre-clinical progress for the last 2 years (2000-2001) and briefly speculate on future prospects for the next 2 in CF gene therapy.

Go Back to Publications     View on Pubmed (12364999)    View on Google Scholar