摘要:
Methods are provided for obtaining measurable levels of a protein, nucleic acid molecule, or enzymatic product in a bodily fluid or cells of a human, comprising the step of administering to a human a recombinant retroviral preparation having a titer on HT1080 cells of greater than 105 cfu/ml, wherein the recombinant retroviral preparation is capable of directing the expression of a protein, nucleic acid molecule, or enzyme which generates an enzymatic product, such that measurable levels of the protein, nucleic acid molecule, or enzymatic product may be obtained in the bodily fluid or cells of the human.
摘要:
Gene delivery vectors, such as, for example, recombinant FIV vectors, and methods of using such vectors are provided for use in treating or preventing retinal diseases of the eye and diseases of the brain associated with lysosomal storage disorders.
摘要:
The present invention provides a method for targeting a gene delivery vehicle to a selected cell type in a warm-blooded animal, comprising the step of administering to a warm-blooded animal a targeting element coupled to a first high affinity molecule of a high affinity binding pair, the coupled targeting element being capable of specifically binding to a selected cell type in the warm-blooded animal, and administering to the animal a gene delivery vehicle coupled to a second high affinity molecule of the high affinity binding pair, the second high affinity molecule being capable of specifically binding to the first high affinity molecule, such that the gene delivery vehicle is targeted to the selected cell type upon administration.
摘要:
The present application discloses the incorporation of specific membrane-associated proteins into enveloped vector particles to provide an altered host range for these viruses. As a model system, the incorporation of the VSV G protein into Moloney murine leukemia virus (MoMLV)-based retroviral vectors is disclosed. The application discloses methods of introducing genes using modified viruses, and the modified viruse's associated nucleic acid, proteins and cell lines. The methods of introducing genes are disclosed to be useful in a variety of gene therapy methods.
摘要:
The present invention provides compositions and methods for treatment of solid tumors with gene therapy utilizing recombinant viral vectors that express polypeptides which selectively initiate irreversible coagulation of blood in the tumor vasculature, inhibit tumor neovascularization, are capable of activating a non-toxic agent into a toxic agent within the tumor vascular wall causing destruction of the vascular bed and absorb or metabolize nutrients in the blood being supplied to the tumor. The production of these polypeptides by transduced cells in or adjacent to the blood vessels of the tumor result in the death of tumor cells.
摘要:
Expression vectors are provided which have a tightly controllable promoter which directs the expression of a fusogenic protein. Also provided are nucleic acid molecules which encode toxic of fusogenic proteins, wherein one or more cysteine residues in the nucleic acid molecule has been altered to an opal termination codon.
摘要:
Compositions and methods for the efficient ex vivo introduction of nucleic acid into T cells, non-dividing cells, and cells resistant to standard transduction techniques mediated by high titer recombinant retroviral preparations is described. The recombinant vector constructs carried by the recombinant retrovirus particles code for the production of one or more desired gene products from one or more corresponding genes of interest, at least one of the gene products having a therapeutic application. Upon re-introduction into a patient, the transduced cells produce a desired gene product in an amount sufficient to treat a particular disease state.
摘要:
Compositions and methods are provided for directing the position-specific integration of a vector construct encoding one or more desired genes into a specific region of a target eukaryotic genome. Such compositions and methods are useful for performing somatic and germ cell gene therapy. Among the benefits provided by position-specific integration are more uniform levels of expression of a desired gene(s) and substantial elimination of the potential for insertional mutagenesis.
摘要:
Compositions and methods for the efficient ex vivo introduction of nucleic acid into hematopoietic stem cells mediated by recombinant retrovirus particles is described. The recombinant vector constructs carried by the recombinant retrovirus particles code for the production of a desired gene product having a therapeutic application from a gene of interest. Upon re-introduction into a patient, the transduced hematopoietic stem cells produce a desired gene product in an amount sufficient to treat a particular disease state.
摘要:
Lentiviral vectors are provided comprising a 5' lentiviral LTR, a tRNA binding site, a packaging signal, a promoter operably linked to one or more genes of interest, an origin of second strand DNA synthesis and a 3' lentiviral LTR, wherein the lentiviral vector contains a nuclear transport element that is not RRE. Also provided are expression cassettes and packaging cell lines for producing lentiviral vector particles.