MINI-ADENOVIRAL VECTOR AND METHODS OF USING SAME

摘要

The present invention provides a method for treating a disorder such as hemophilia. A method of treating in a mammal by administering recombinant virus virions comprising a nucleotide sequence having an adenoviral inverted terminal repeat fusion sequence, packaging signal, a transcriptional control region, and a nucleic acid encoding a therapeutic protein such as FVIII. In addition, the DNA molecule does not encode an adenoviral protein. It is preferred that the virions be adminstered to the mammal under conditions that result in the expression of the therapeutic protein at a level that provides a therapeutic effect in said mammal. In addition, the virions are administered with immunosuppressive agents.