Current Opinion In Molecular Therapeutics
Curr Opin Mol Ther. 2001 Aug;3(4):385-9.
Cystic fibrosis (CF) is a monogenic disorder and is therefore a good candidate for gene therapy. Initial clinical trials provided proof-of-principle for gene transfer to the airways, but efficiency was low and likely to be insufficient for clinical benefit. Here, we review the progress in CF gene therapy over the last 12 months, including recent advances in viral and non-viral gene transfer agents and novel strategies, such as RNA repair and stem cell gene therapy.