Transkaryotic preclinical data

The Cambridge, Mass., company published data showing stable, life-long production of human growth hormone in mice given cells transfected with the human gene. The company said it was the first report of lifetime expression of a protein in any gene therapy system.

As described in the Proceedings of the National Academy of Sciences, transfection of rabbit dermal cells with plasmids bearing the gene was carried out by electroporation. Then a single fibroblast producing the protein was chosen and cloned. ...