Abstract: The invention provides improved gene therapy vectors, compositions, and methods.

Abstract: The invention provides improved gene therapy vectors, compositions, and methods.

Abstract: The invention provides improved gene therapy vectors, compositions, and methods.

Abstract: The invention generally provides methods for producing recombinant AAV viral particles using cells grown in suspension. The invention provides recombinant AAV particles for use in methods for delivering genes encoding therapeutic proteins, and methods for using the recombinant AAV particles in gene therapy.

Abstract: The present invention provides compositions comprising retroviral vectors, transduced cells, and methods of using the same for gene therapy. In particular, the present invention relates to lentiviral vectors and cells transduced with those vectors to provide gene therapy to subjects having an adrenoleukodystrophy and/or adrenomyeloneuropathy.

Abstract: The invention generally provides methods for producing recombinant AAV viral particles using cells grown in suspension. The invention provides recombinant AAV particles for use in methods for delivering genes encoding therapeutic proteins, and methods for using the recombinant AAV particles in gene therapy.

Abstract: The present invention provides compositions comprising retroviral vectors, transduced cells, and methods of using the same for gene therapy. In particular, the present invention relates to lentiviral vectors and cells transduced with those vectors to provide gene therapy to subjects having an adrenoleukodystrophy and/or adrenomyeloneuropathy.

Abstract: The present invention provides compositions comprising retroviral vectors, transduced cells, and methods of using the same for gene therapy. In particular, the present invention relates to lentiviral vectors and cells transduced with those vectors to provide gene therapy to subjects having an adrenoleukodystrophy and/or adrenomyeloneuropathy.

Abstract: The present invention provides compositions comprising retroviral vectors, transduced cells, and methods of using the same for gene therapy. In particular, the present invention relates to lentiviral vectors and cells transduced with those vectors to provide gene therapy to subjects having an adrenoleukodystrophy and/or adrenomyeloneuropathy.

Abstract: The present invention provides compositions comprising retroviral vectors, transduced cells, and methods of using the same for gene therapy. In particular, the present invention relates to lentiviral vectors and cells transduced with those vectors to provide gene therapy to subjects having an adrenoleukodystrophy and/or adrenomyeloneuropathy.

Abstract: The invention generally provides methods for producing recombinant AAV viral particles using cells grown in suspension. The invention provides recombinant AAV particles for use in methods for delivering genes encoding therapeutic proteins, and methods for using the recombinant AAV particles in gene therapy.

Abstract: The present invention provides compositions comprising retroviral vectors, transduced cells, and methods of using the same for gene therapy. In particular, the present invention relates to lentiviral vectors and cells transduced with those vectors to provide gene therapy to subjects having an adrenoleukodystrophy and/or adrenomyeloneuropathy.

Abstract: The invention generally provides methods for producing recombinant AAV viral particles using cells grown in suspension. The invention provides recombinant AAV particles for use in methods for delivering genes encoding therapeutic proteins, and methods for using the recombinant AAV particles in gene therapy.

Abstract: The present invention concerns a method of identifying genetically modified mammalian cells using a mutated protein-tyrosine kinase receptor (PTKR) as a selectable marker in mammalian cells. Particularly preferred mutated PTKR selective markers are mutated epidermal growth factor receptor (EGFR) family members, and muscle specific tyrosine kinase receptor (MuSK-R) family members. Further a method for the immunoselection of transduced mammalian cells is disclosed comprising retrovirally transducing mammalian cells with a nucleic acid sequence encoding a mutated EGFR, incubating the transduced cells with a marked antibody which recognizes and binds specifically to the mutated PTKR, and identifying the marked transduced cells.

Abstract: Novel antisense sequences to the unspliced or single spliced portions of mRNA transcript from HIV-1 provirus, optionally co-expressed with an inhibitory transdominant mutant HIV-1 protein, are found to be useful in the treatment of HIV-1 infection.

Abstract: The present invention concerns a method of using a muscle specific tyrosine kinase receptor molecule (MuSK-R) or a mutated MuSK-R (mMuSK-R) thereof as a selectable marker in mammalian cells, particularly human cells. Preferred markers are mMuSK-Rs incapable of signal transduction and preferably molecules wherein the intracellular domain has been modified by deletion of the signaling region. The invention also relates to a method of identifying genetically modified mammalian cells including introducing a mMuSK-R into a target cell as a selectable marker. Further a method for the immunoselection of transduced mammalian cells is disclosed comprising identifying the transduced cells by incubation of the cells with an antibody which recognizes and binds specifically to a MuSK-R or mMuSK-R thereof.

Abstract: This invention relates to a method of using scaffolding attachment regions (SARs) to increase expression in retrovirally transduced resting cells. This includes gene therapy by introducing into a patient a cellular composition comprising non-immortal human cells transduced with a retroviral vector comprising a DNA SAR element and a heterologous gene operatively linked to an expression control sequence. A particularly preferred SAR is the 5′ SAR of the human interferon &bgr; gene or a fragment thereof.

Abstract: This invention relates to a method of using scaffold attachment regions (SARs) to increase expression in retrovirally transduced resting cells. A particularly preferred SAR is the 5′ SAR of the human interferon &bgr; gene or a fragment thereof having at least 450 base pairs.