Correction of Serum Protein Deficiencies with Recombinant Adenoviral
James N. Higginbotham and Prem Seth
There are numerous inherited and acquired serum protein deficiencies that cause moderate to severe and even life threatening diseases in people. Some of the diseases include α1-antitrypsin deficiency, hemophilias A and B, diabetes mellitus, and anemia. A number of these diseases have been treated by repeated subcutaneous injections of purified recombinant proteins; however, such therapies are generally expensive and inconvenient to the patient or in some cases not enough of the recombinant protein is available for prophylactic treatment. It is important that any serum protein deficiency to be studied have an accurate and reproducible assay by which the patient or physician can monitor serum levels for effective dosing. Because of problems of recombinant protein replacement, much attention has focused on the development of gene-based therapies. The use of adenoviral vectors to deliver genes encoding serum proteins has been extensively reported. Perhaps the most important advantage of adenoviral vectors is the extremely wide range of host cells they can efficiently transduce. The wide tropism of adenoviral vectors allows the use of numerous target tissues or organs like liver, lung, and muscle. Equally important is their independence of cell cycle for efficient gene transfer and expression.