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.
Gene Therapy Progress and Prospects: Cystic Fibrosis
FERRARI S;
2002
Abstract
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.File in questo prodotto:
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