Gene Therapy for Cystic Fibrosis - An UpdateDr Tim W.R. Lee BSc, MB ChB, PhD. Jan 2005.[online] Gene therapy for cystic fibrosis - An update. Regional Paediatric CF Centre, St James’s University Hospital, Leeds, UK. Available from
http://www.cysticfibrosismedicine.comSummary The first gene of mutation responsible for cystic fibrosis was first identified in 1989. Since that time gene therapy has held the promise of a cure for cystic fibrosis, however progress has been much slower than initially hoped. Clinical trials of gene therapy have demonstrated that the corrected CF gene can be successfully expressed in the respiratory tract of subjects with cystic fibrosis, but only for approximately 30 days at best, and at low levels.
Bronchial epithelial cells have a lifetime of approximately 120 days before being replaced, thus to maintain expression of the corrected CF gene repeat dosing of the gene therapy will be necessary, unless lung stem cells can be targeted. Delivery systems include using modified viruses such as adenovirus and adeno-associated virus, or non-viral systems such as liposomes.
This review provides an overview of the basic mechanisms needed to achieve gene therapy, as well as detailing the clinical trials of cystic fibrosis gene therapy to date. Important pre-clinical data and future prospects for gene therapy are discussed.
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