Targeting expression of a transgene to the airway surface epithelium using a ciliated cell-specific promoter

Abstract excerpt

Many of the vectors being investigated for gene therapy utilize viral promoters or promoters from ubiquitously expressed genes (e.g., CMV, beta-actin). These promoters are active in many cell types and generally result in high levels of transgene expression. However, the use of these promoters for gene therapy of cystic fibrosis (CF) may produce undesirable effects by directing high levels of CFTR expression in...

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