Abstract: Mammalian cell lines genetically engineered to be viral resistant, cell culture systems comprising agents that inhibit viral entry into or translocation within cells, and methods of using said cell lines and/or said cell culture systems to reduce or prevent viral contamination of biologic production systems.

Abstract: The present description relates to an inhibitory RNA molecule, comprising an oligonucleotide that selectively knocks down expression a Nanog pseudogene expressed in many human cancers, a replicating viral vector capable of encoding such inhibitory RNA molecule, pharmaceutical compositions comprising said vector, and methods of treating cancer by administration of said pharmaceutical composition.

Abstract: The present description relates to an inhibitory RNA molecule, comprising an oligonucleotide that selectively knocks down expression a Nanog pseudogene expressed in many human cancers, a replicating viral vector capable of encoding such inhibitory RNA molecule, pharmaceutical compositions comprising said vector, and methods of treating cancer by administration of said pharmaceutical composition.

Abstract: The present description relates to an inhibitory RNA molecule, comprising an oligonucleotide that selectively knocks down expression a Nanog pseudogene expressed in many human cancers, a replicating viral vector capable of encoding such inhibitory RNA molecule, pharmaceutical compositions comprising said vector, and methods of treating cancer by administration of said pharmaceutical composition.

Abstract: The present description relates to an inhibitory RNA molecule, comprising an oligonucleotide that selectively knocks down expression a Nanog pseudogene expressed in many human cancers, a replicating viral vector capable of encoding such inhibitory RNA molecule, pharmaceutical compositions comprising said vector, and methods of treating cancer by administration of said pharmaceutical composition.

Abstract: The present invention describes the use of pre-trans-splicing molecules (PTMs) to reprogram human normal and diseased somatic cells into pluripotent stem cells using spliceosome-mediated RNA trans-splicing. More specifically, the present invention describes the use of the SMaRT™ technology to repair or reprogram the newly induced diseased pluripotent stem cells.

Abstract: The present invention provides for lentiviral vectors for vaccine delivery comprising a 5? long terminal repeat (LTR) and a 3? LTR, an integrated nucleic acid sequence, wherein the integrated nucleic acid sequence is expressed by the 5? LTR; and a nucleic acid sequence encoding functional REV coding sequence and a rev response element (RRE)-containing sequence, wherein the RRE-containing sequence is located upstream of the REV coding sequence, and wherein transcription of said first nucleic acid sequence and said second nucleic acid sequence is driven by said 5? LTR. Also provided for are pharmaceutical compositions, methods of making and using the lentiviral vectors of the present invention.

Abstract: The present invention encompasses replication deficient or a replication competent adenoviral vectors which may comprise moieties covering and shielding the vector from the effects of humoral immune responses, as well as a method of constructing and using such vectors. The preferred viral constructs may incorporate the shielding moieties into the pIX coat protein of the adenovirus vectors. The invention also provides recombinant viral vectors with both shielding and specific targeting abilities. Preferably, the viral vector may comprise a nucleic acid sequence, which codes for therapeutically important genes. Methods for treating of a host with an effective amount of adenovirus vector of the present invention are also provided.

Abstract: The present invention provides DC-SIGN-targeted recombinant adenoviral vectors and methods of using these vectors to transduce immature dendritic cells. More specifically, these vectors employ a two-component targeting moiety which may contain an adenoviral fiber protein that may comprise an immunoglobulin-binding domain and an anti-DC-SIGN antibody as a targeting ligand.

Abstract: The present invention provides a recombinant adenovirus vector targeted by zipper peptides. The vector contains a fiber protein modified by insertion of a first zipper peptide that can crosslink to a fusion protein comprising a second zipper peptide and a targeting ligand. Binding of the first zipper peptide to the second zipper peptide forms a targeted vector: ligand complex, thereby targeting the adenovirus vector to a cell that expresses a cell surface molecule that binds to said targeting ligand.

Abstract: The utility of adenovirus vectors (Ad) for gene therapy is restricted by their inability to selectively transduce disease-affected tissues. This limitation may be overcome by the derivation of vectors capable of interacting with receptors specifically expressed in the target tissue. Previous attempts to alter Ad tropism by genetic modification of the Ad fiber have had limited success due to structural conflicts between the fiber and the targeting ligand. The present invention presents a strategy to derive an Ad vector with enhanced targeting potential by a radical replacement of the fiber protein in the Ad capsid with a chimeric molecule containing a heterologous trimerization motif and a stabilized scFv ligand.

Abstract: The present invention provides a targeted recombinant adenovirus vector expressing a fiber protein modified by insertion of an immunoglobulin-binding domain that can crosslink to a fusion protein comprising a targeting ligand and an immunoglobulin Zc domain. Interaction between the immunoglobulin-binding domain and the Zc domain results in a targeted vector::ligand complex, thereby targeting the adenovirus vector to a cell that expresses a cell surface molecule that binds to said targeting ligand.

Abstract: The present invention provides a recombinant adenovirus vector targeted by zipper peptides. The vector contains a fiber protein modified by insertion of a first zipper peptide that can crosslink to a fusion protein comprising a second zipper peptide and a targeting ligand. Binding of the first zipper peptide to the second zipper peptide forms a targeted vector: ligand complex, thereby targeting the adenovirus vector to a cell that expresses a cell surface molecule that binds to said targeting ligand.

Abstract: The present invention provides a targeted recombinant adenovirus vector expressing a fiber protein modified by insertion of an immunoglobulin-binding domain that can crosslink to a fusion protein comprising a targeting ligand and an immunoglobulin Fc domain. Interaction between the immunoglobulin-binding domain and the Fc domain results in a targeted vector::ligand complex, thereby targeting the adenovirus vector to a cell that expresses a cell surface molecule that binds to said targeting ligand.

Abstract: The present invention provides adenoviral vectors (Ad) that incorporate multiple distinct capsid modifications in a single virus particle, resulting in Ad that have improved gene delivery capacities and/or vector function. The capsid modifications include (a) addition of a heterologous targeting ligand; (b) a fiber shaft with altered length; (c) capsid modification that alters the charge of the adenoviral vector; and (d) replacing the capsid proteins with capsid proteins from another serotype.

Abstract: The present invention provides adenoviral vectors (Ad) that incorporate multiple distinct capsid modifications in a single virus particle, resulting in Ad that have improved gene delivery capacities and/or vector function.