Abstract: A vector and a method are provided for delivering a nucleic acid to a nervous system cell. The vector includes a first nucleic acid, a second nucleic acid, inverted terminal repeats of adeno-associated virus, and a tetracycline-off regulatable promoter system that includes a first promoter operably linked to the first nucleic acid and a second promoter operably linked to the second nucleic acid. The promoters drive expression in opposite directions and away from the inverted terminal repeats. The method includes providing a recombinant adeno-associated viral (rAAV) vector and administering the vector to a nervous system cell. Expression of a product from the first nucleic acid is regulatable by the promoter system.

Abstract: A vector and a method are provided for delivering a nucleic acid to a nervous system cell. The vector includes a first nucleic acid, a second nucleic acid, inverted terminal repeats of adeno-associated virus, and a tetracycline-off regulatable promoter system that includes a first promoter operably linked to the first nucleic acid and a second promoter operably linked to the second nucleic acid. The promoters drive expression in opposite directions and away from the inverted terminal repeats. The method includes providing a recombinant adeno-associated viral (rAAV) vector and administering the vector to a nervous system cell. Expression of a product from the first nucleic acid is regulatable by the promoter system.

Abstract: A vector and a method are provided for delivering a nucleic acid to a nervous system cell. The vector includes a first nucleic acid, a second nucleic acid, inverted terminal repeats of adeno-associated virus, and a tetracycline-off regulatable promoter system that includes a first promoter operably linked to the first nucleic acid and a second promoter operably linked to the second nucleic acid. The promoters drive expression in opposite directions and away from the inverted terminal repeats. The method includes providing a recombinant adeno-associated viral (rAAV) vector and administering the vector to a nervous system cell. Expression of a product from the first nucleic acid is regulatable by the promoter system.

Abstract: The present invention pertains to a method for efficiently introducing exogenous genes into stem cells, particularly human stem cells. The method optionally includes the steps of inducing the proliferation of target cells by pre-stimulation with cytokines and/or growth factors, followed by incubating these cells with RD114-pseudotyped vector particles. In a specific embodiment, the vector particles are retronectin-immobilized or ultracentrifugation-concentrated retroviral vector particles pseudotyped with the feline endogenous retrovirus (RD114) envelope protein. The present invention further discloses a method for somatic gene therapy, which can be used for various therapeutic applications and involves introducing a gene of interest contained within the retroviral genome into human repopulating stem cells followed by introducing these cells into a human host.

Abstract: A method of transferring at least one DNA sequence into cells by transducing the cells, in vivo or ex vivo, with a modified adenovirus. The adenovirus, prior to modification, is of a first serotype. In the modified adenovirus, at least a portion of the fiber, and in particular the head portion, is removed from the adenovirus of the first serotype and replaced with a portion, in particular the head portion, of the fiber of an adenovirus of a second serotype. Such method is useful in transducing cells which may be refractory to the adenovirus of the first serotype, yet include a receptor which binds to the head portion of the fiber of the adenovirus of the second serotype.

Abstract: A method of transferring at least one DNA sequence into cells by transducing the cells, in vivo or ex vivo, with a modified adenovirus. The adenovirus, prior to modification, is of a first serotype. In the modified adenovirus, at least a portion of the fiber, and in particular the head portion, is removed from the adenovirus of the first serotype and replaced with a portion, in particular the head portion, of the fiber of an adenovirus of a second serotype. Such method is useful in transducing cells which may be refractory to the adenovirus of the first serotype, yet include a receptor which binds to the head portion of the fiber of the adenovirus of the second serotype.

Abstract: A method of transferring at least one DNA sequence into cells by transducing the cells, in vivo or ex vivo, with a modified adenovirus. The adenovirus, prior to modification, is of a first serotype. In the modified adenovirus, at least a portion of the fiber, and in particular the head portion, is removed from the adenovirus of the first serotype and replaced with a portion, in particular the head portion, of the fiber of an adenovirus of a second serotype. Such method is useful in transducing cells which may be refractory to the adenovirus of the first serotype, yet include a receptor which binds to the head portion of the fiber of the adenovirus of the second serotype.

Abstract: The present invention pertains to a method for efficiently introducing exogenous genes into stem cells, particularly human stem cells. The method optionally includes the steps of inducing the proliferation of target cells by pre-stimulation with cytokines and/or growth factors, followed by incubating these cells with RD114-pseudotyped vector particles. In a specific embodiment, the vector particles are retronectin-immobilized or ultracentrifugation-concentrated retroviral vector particles pseudotyped with the feline endogenous retrovirus (RD114) envelope protein. The present invention further discloses a method for somatic gene therapy, which can be used for various therapeutic applications and involves introducing a gene of interest contained within the retroviral genome into human repopulating stem cells followed by introducing these cells into a human host.

Abstract: The field of the invention is recombinant packaging and producer cell lines for producing infectious retroviral vectors. The invention more specifically relates to the generation of pseudotyped retroviral vectors with a broad host range which can be produced at high titers in specially constructed packaging cell lines. Most specifically, the invention relates to the generation of pseudotyped retroviral vectors having vesicular stomatitis virus-G protein (VSV-G) as the membrane-associated viral envelope protein.

Abstract: A retroviral vector which includes a nucleic acid sequence encoding a retroviral envelope. The nucleic acid sequence encoding a retroviral envelope includes a first nucleic acid sequence encoding a first envelope portion which is a portion of MCF viral gp 70 protein, a nucleic acid sequence which encodes xenotropic envelope, a nucleic acid sequence which encodes an amphotropic envelope portion, and a nucleic acid sequence which encodes p15E protein. Such retroviral envelopes encoded by such nucleic acid sequence may be included in infectious viral particles. The infectious viral particles also may include gene(s) encoding therapeutic agents, and thus may be used in gene therapy.