New adenoviral vector useful for gene therapy

摘要

An adenoviral (Ad) transfer vector (A) for gene transport of a DNA sequence is new and is produced from an Ad plasmid that no longer expresses a native Ad protein. An adenoviral (Ad) transfer vector (A) for gene transport of a DNA sequence is produced from an Ad plasmid that no longer expresses a native Ad protein and: (1) includes a first DNA sequence having the left inverted terminal repeat (ITR) and a packaging signal of wild-type Ad (serotype 5); (2) includes a second Ad sequence containing the right ITR; (3) includes cleavage sites for restriction enzymes that are not present in the marker and/or therapeutic genes inserted between the Ad sequences, and (4) the ITRs are flanked by cleavage sites for FseI so that excision of the Ad part of the transfer vector is possible. Independent claims are also included for the following: (1) cDNA (I) encoding a B-domain truncated factor VIII (II) containing amino acids 1-852 and 1524-2332 of human factor VIII; (2) (II); and (3) a composition containing (A) plus an agent (B) with anti-CD4 activity.