Abstract: The production of genetically modified animals, in which the genetic modifications are engineered in somatic cells cultured in vitro by gene targeting, is described. Genetically modified cells may then used as nuclear donors to produce, inter alia, live animals. The methods described can also be used to validate loci in animal chromosomes which are suitable sites for transgene addition to cells.

Abstract: The present invention provides a method for treating a disorder such as hemophilia. A method of treating hemophilia in a mammal by administering recombinant virus virions comprising a nucleotide sequence having an adenoviral inverted terminal repeat fusion sequence, a 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 administered 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.

Abstract: This invention is related to adenoviral (Ad) vectors and their applications in the field of genetic medicine, including gene transfer, gene therapy, and gene vaccination. More specifically, this invention is related to the Ad vectors that carry the minimal cis-element of the Ad genome (mini-Ad vector) and are capable of delivering transgenes and/or heterologous DNA up to 36 kb. The generation and propagation of the mini-Ad vectors require trans-complementation of a packaging-attenuated and replication-defective helper Ad (helper) in an Ad helper cell line.

Abstract: The invention provides reagents and methods for highly efficient generation of adenoviral vectors by homologous recombination. The present invention provides unique shuttle vectors and an improved methodology for co-transfection of a shuttle vector and a helper plasmid into 293 cells to generate E1-deleted, E1/E3-deleted, E1/E2a/E3-deleted or E1/E3/E4/protein IX-deleted adenoviral vectors.

Abstract: The production of genetically modified animals, in which the genetic modifications are engineered in somatic cells cultured in vitro by gene targeting, is described. Genetically modified cells may then used as nuclear donors to produce, inter alia, live animals. The methods described can also be used to validate loci in animal chromosomes which are suitable sites for transgene addition to cells.

Abstract: The production of genetically modified animals, in which the genetic modifications are engineered in somatic cells cultured in vitro by gene targeting, is described. Genetically modified cells may then used as nuclear donors to produce, inter alia, live animals. The methods described can also be used to validate loci in animal chromosomes which are suitable sites for transgene addition to cells.