Published in Gene Therapy Weekly, September 6th, 2001
In order for gene therapy to effectively treat cystic fibrosis, it is critical that airway epithelial cells are transduced with the therapeutic gene. Up to now, many researchers have been working with the adeno-associated virus type 2 (AAV2) vector. Difficulties lie in the fact that the apical surfaces of airway epithelial cells are highly resistant to AAV2 transfection. Attempts to improve transfection efficiency by increasing vector dose and "perturbing the integrity of the epithelium"...
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