"Gene Therapy for Cystic Fibrosis using Adenovirus Vectors."

According to the authors' abstract of an article published in M S - Medecine Sciences, "Mutations in the CFTR gene are responsible for the clinical manifestations of cystic fibrosis (CF). Since approximately 95% of the morbidity and mortality related to the disease arise from pulmonary complications, the primary target for gene therapy of CF is the respiratory epithelium, in which reversal of tile CF phenotype can be envisaged. In the past few years, CF has thus emerged as an early experimental model for human gene therapies utilizing the first generation (E1-deleted) recombinant adenoviruses. The efforts undertaken to implement a safe and efficient transfer of the CFTR gene to the...

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