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Previous Volume 352:1146-1147 March 17, 2005 Number 11 Next

	Since this article has no abstract, we have provided an extract of the first 100 words of the full text and any section headings.

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All gene-therapy strategies require three elements: a vehicle for gene delivery, a therapeutic gene (sometimes called the transgene), and a physiologically relevant target cell to which the gene is delivered. For some diseases, the choice of target cell is strictly limited — for example, to hematopoietic cells in the case of hemoglobinopathies and to cells in the respiratory tract in the case of the lung disease of cystic fibrosis. For other diseases, such as plasma protein deficiencies (e.g., hemophilia or erythropoietin-responsive anemia), there is latitude in the choice of target cell, as long as the transgene product can be secreted . . . [Full Text of this Article]

Source Information

From the Howard Hughes Medical Institute, the Children's Hospital of Philadelphia, and the University of Pennsylvania — all in Philadelphia.

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