Abstract: Nucleases and methods of using these nucleases for modification of an HPRT locus and for increasing the frequency of gene modification at a targeted locus and clones and for generating animals.

Abstract: This disclosure provides a retroviral replicating vector for gene delivery comprising a therapeutic cassette containing at least one mini-promoter linked to a gene to be expressed.

Abstract: Nucleases and methods of using these nucleases for modification of an HPRT locus and for increasing the frequency of gene modification at a targeted locus and clones and for generating animals.

Abstract: A recombinant replication competent retrovirus for gene deliver and gene therapy is provided. The recombinant retrovirus has a heterologous nucleic acid sequence, a sequence encoding a cell- or tissue-specific ligand or a sequence for transcriptional targeting, or a combination of both a cell- or tissue-specific ligand and a cell- or tissue-specific transcriptional targeting sequence.

Abstract: A method of radiation-free hematopoietic stem cell (HSC) transplantation comprises administering to a mammalian subject one or two doses of 2 to 10 mg/kg body weight of a purine base analog, such as 6TG as a pre-conditioning step. The method further comprises engrafting into the subject hypoxanthine-guanine phosphoribosyltransferase (HPRT)-deficient donor HSCs within 48 to 72 hours of the pre-conditioning step; and administering to the subject about 1 to 5 mg/kg of the purine base analog every two to four days for two to eight weeks following the engrafting step. The method is performed in the absence of pre-conditioning via radiation. The subject is therefore not treated with myeloablative radiation in preparation for transplantation, and thus the subject is free of myeloablative radiation-induced toxicity.

Abstract: A recombinant replication competent retrovirus for gene deliver and gene therapy is provided. The recombinant retrovirus has a heterologous nucleic acid sequence, a sequence encoding a cell- or tissue-specific ligand or a sequence for transcriptional targeting, or a combination of both a cell- or tissue-specific ligand and a cell- or tissue-specific transcriptional targeting sequence.

Abstract: A recombinant replication competent retrovirus for gene deliver and gene therapy is provided. The recombinant retrovirus has a heterologous nucleic acid sequence, a sequence encoding a cell- or tissue-specific ligand or a sequence for transcriptional targeting, or a combination of both a cell- or tissue-specific ligand and a cell- or tissue-specific transcriptional targeting sequence.

Abstract: A method can include making one or more compositions for altering allogeneic cells of a human donor for a human recipient by identifying at least one mismatch in an HLA protein between the human donor and the human recipient; determining a consensus conserved nucleic acid sequence among nucleic acid sequences encoding a domain having the mismatch or among domains having a plurality of mismatches; and forming at least one of the one or more compositions by constructing a virus vector for expressing a sequence targeting the consensus conserved nucleic acid sequence, which when expressed in cells functions as a negative modulator for nucleic acid encoding the domain having the mismatch or the domains having the plurality of mismatches.

Abstract: Vector compositions and methods are provided for transfecting donor cell populations and tissues for grafts, for reducing expression of mismatched proteins, thereby reducing immunogenicity of the grafted cells and tissues in a recipient subject.

Abstract: A recombinant replication competent retrovirus for gene deliver and gene therapy is provided. The recombinant retrovirus has a heterologous nucleic acid sequence, a sequence encoding a cell- or tissue-specific ligand or a sequence for transcriptional targeting, or a combination of both a cell- or tissue-specific ligand and a cell- or tissue-specific transcriptional targeting sequence.

Abstract: Methods and kits are provided for determining of immunoglobulin isotypes and subclasses in a subject. In general the subject is a human who is a transplant candidate recipient or recipient, has allergies, or has an autoimmune disease. The method involves analyzing a sample of a body fluid of a transplant candidate or recipient, allergy patient or autoimmune disease sufferer and correlating the relative amounts of each immunoglobulin isotype and subtype, such that the distribution of isotypes and subtypes is an indication of success of the transplant in the candidate and recipient or the prognosis of the autoimmune disease.

Abstract: The present invention demonstrates that VSV-G-pseudotyped lentivirus vectors efficiently transduce AEC in primary culture and in vivo with transduction favored by virus application from the apical side. Transduction efficiency in AEC increased with increasing MOI and greatly exceeded that achieved with a similarly pseudotyped MLV retrovirus vector. The present invention also demonstrates the successful in vivo transfer of genes through lentivirus vector transduction. Mammals injected with lentivirus vector via the trachea expressed the reporter protein in alveolar epithelial cells within 48 to 72 hours after infection.

Abstract: The present invention provides a plasmid encoding a replication-competent virus for use in therapy more particularly for use in the treatment of a cell proliferative disease, an immunological disease, a neuronal disorder, an acquired infection and inflammation as well as formulations comprising such plasmids together with a transfection agent.

Abstract: The present invention demonstrates that VSV-G-pseudotyped lentivirus vectors efficiently transduce AEC in primary culture and in vivo with transduction favored by virus application from the apical side. Transduction efficiency in AEC increased with increasing MOI and greatly exceeded that achieved with a similarly pseudotyped MLV retrovirus vector. The present invention also demonstrates the successful in vivo transfer of genes through lentivirus vector transduction. Mammals injected with lentivirus vector via the trachea expressed the reporter protein in alveolar epithelial cells within 48 to 72 hours after infection.

Abstract: Vector compositions and methods are provided for transfecting donor cell populations and tissues for grafts, for reducing expression of mismatched proteins, thereby reducing immunogenicity of the grafted cells and tissues in a recipient subject.

Abstract: A recombinant replication competent retrovirus for gene deliver and gene therapy is provided. The recombinant retrovirus has a heterologous nucleic acid sequence, a sequence encoding a cell- or tissue-specific ligand or a sequence for transcriptional targeting, or a combination of both a cell- or tissue-specific ligand and a cell- or tissue-specific transcriptional targeting sequence.

Abstract: A recombinant replication competent retrovirus for gene deliver and gene therapy is provided. The recombinant retrovirus has a heterologous nucleic acid sequence, a sequence encoding a cell- or tissue-specific ligand or a sequence for transcriptional targeting, or a combination of both a cell- or tissue-specific ligand and a cell- or tissue-specific transcriptional targeting sequence.

Abstract: The invention discloses hybrid vectors for delivering genes or other nucleic acids into mammalian cells. The hybrid vectors of the invention contain both a helper dependent adenoviral portion and a second portion derived from a transposon. Such vectors provide efficient transduction of quiescent cells and provide for stable integration of the gene to be delivered.

Abstract: The invention discloses hybrid vectors for delivering genes or other nucleic acids into mammalian cells. The hybrid vectors of the invention contain both a helper dependent adenoviral portion and a second portion derived from either a replication incompetent retrovirus or from a transposon. Such vectors provide efficient transduction of quiescent cells and provide for stable integration of the gene to be delivered.

Abstract: The invention discloses hybrid vectors for delivering genes or other nucleic acids into mammalian cells. The hybrid vectors of the invention contain both a helper dependent adenoviral portion and a second portion derived from a transposon. Such vectors provide efficient transduction of quiescent cells and provide for stable integration of the gene to be delivered.