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Gene therapy is being investigated in the treatment of lung-related aspects of the genetic disease, Cystic fibrosis (CF). Clinical studies have demonstrated CF transmembrane conductance regulator (CFTR) expression in the airways of adults with CF using a variety of gene transfer agents. In utero gene therapy is an alternative approach that facilitates vector transduction of rapidly expanding populations of target cells while avoiding immune recognition of the vector. In CF, in utero gene transfer could potentially delay the onset of disease symptoms in childhood and compensate for the role, if any, that CFTR plays in the developing organs. Previously published studies have suggested that transient expression of CFTR in utero was sufficient to rescue the fatal intestinal defect in S489X Cftr(tm1Unc)/Cftr(tm1Unc) knockout mice. We replicated these studies using an identical CFTR-expressing adenoviral vector and CF mouse strain in sufficiently large numbers to provide robust Kaplan-Meier survival data. Although each step of the procedure was carefully controlled and vector-specific CFTR expression was confirmed in the fetal organs after treatment, there was statistically no significant improvement in the survival of mice treated in utero with AdCFTR, compared with contemporaneous control animals.

Original publication

DOI

10.1038/mt.2008.25

Type

Journal article

Journal

Mol Ther

Publication Date

05/2008

Volume

16

Pages

812 - 818

Keywords

Adenoviridae, Amniotic Fluid, Animals, Cystic Fibrosis, Cystic Fibrosis Transmembrane Conductance Regulator, Female, Gene Expression Regulation, Genetic Therapy, Genetic Vectors, Humans, Mice, Mice, Inbred C57BL, Mice, Knockout, Pregnancy, Pregnancy, Animal