Abstract:
A rapid method for modifying a viral capsid or envelope protein with a polyethylene glycol (PEG) is described. Also provided are methods of delivering a molecule using the PEG-modified viruses of the invention. Compositions containing the PEG-modified viruses of the invention, are characterized by improved gene expression, reduced neutralizing antibody and CTL production. Also provided are viral compositions having enhanced physical stability, in which the viruses are lyophilized in a formulation having a 1:1 ratio of sucrose and mannitol are provided.
Abstract:
Novel cell lines useful for trans-complementing E1-deleted adenoviral vectors are described. The cell lines are capable of providing high yields of E1-deleted adenoviral vectors in the absence of replication-competent adenovirus over multiple passages.
Abstract:
Genetically engineered or transduced hepatocytes which express genetic material of interest introduced or incorporated into them, as well as methods of producing, transplanting and using the genetically engineered hepatocytes. The genetic material of interest can be incorporated through use of a vector, such as a recombinant retrovirus, which contains the genetic material of interest, or by other method.
Abstract:
A replication defective recombinant adenovirus is provided which contains a complete deletion of its E1 gene and at least a partial deletion of its E3 gene, said virus containing in the site of the E1 deletion a sequence comprising a non-adenovirus promoter directing the replication and expression of DNA encoding a heterologous protein from a disease-causing agent, which, when administered to a mammal in said recombinant virus, elicits a substantially complete protective immune response against the agent. Pharmaceutical and veterinary products containing the recombinant adenovirus are provided.
Abstract:
The present invention provides a hybrid vector construct which comprises a portion of an adenovirus, 5′ and 3′ ITR sequences from an AAV, and a selected transgene. Also provided is a hybrid virus linked via a polycation conjugate to an AAV rep gene to form a single particle. These trans-infection particles are characterized by high titer transgene delivery to a host cell and the ability to stably integrate the transgene into the host cell chromosome. Also disclosed is the use of the hybrid vectors and viruses to produce large quantities of recombinant AAV.
Abstract:
Endothelial cells transduced with genetic material encoding a polypeptide or protein of interest and, optionally, a selectable marker, as well as methods for making and using the transduced endothelial cells are disclosed. Such endothelial cells are useful in improving the performance of vascular grafts and in delivering the encoded polypeptide or protein, such as an enzyme, a hormone, a receptor or a drug, to an individual.
Abstract:
The present invention provides a hybrid vector construct which comprises a portion of an adenovirus, 5' and 3' ITR sequences from an AAV, and a selected transgene. Other hybrid vectors form a polycation conjugate and incorporate an AAV rep gene in a single particle. These hybrid virus vectors are characterized by high titer transgene delivery to a host cell and the ability to stably integrate the transgene into the host cell chromosome. Also disclosed is the use of the hybrid vectors to produce large quantities of recombinant AAV.
Abstract:
A non-human animal characterized by a human airway, the human airway being open-ended. The human airway is characterized by cells derived from the human respiratory system. The non-human animal and the human airway it carries are useful as models of human airways, particularly diseased human airways, for example, human CF airways. They are also useful to predict the efficacy or toxicity of agents and therapies on human airways, particularly on diseased human airways, for example, human CF airways.
Abstract:
The invention provides a recombinant viral vector comprising the DNA of, or corresponding to, at least a portion of the genome of an adenovirus, which portion is capable of infecting a hepatic cell; and a human VLDL receptor gene operatively linked to regulatory sequences directing its expression. The vector is capable of expressing the normal VLDL receptor gene product in hepatic cells in vivo or in vitro. This viral vector is useful in the treatment of metabolic disorders caused by the accumulation of LDL in plasma, such as familial hypercholesterolemia or familial combined hyperlipidemia.