Abstract: The present invention provides a bovine adeno-associated virus (BAAV) virus and vectors and particles derived therefrom. In addition, the present invention provides methods of delivering a nucleic acid to a cell using the BAAV vectors and particles.

Abstract: The present invention relates to conditionally replicating viruses or pairs of viruses containing a gene switch that is activatable by transient heat or other proteotoxic stress in the presence or absence of a small molecule regulator. The gene switch controls the expression of a gene for a protein required for efficient viral replication and may also control the activity of a passenger gene.

Abstract: A targeted drug delivery nanocarrier and a method of forming the same is disclosed herein. The targeted drug delivery nanocarrier includes a plurality of amphipathic molecules forming a carrier particle having a plurality of drug molecules contained therein. A targeted landscape phage protein assembly is complexed to the carrier particle preferably using the unique method disclosed herein. The targeted landscape phage protein assembly displays a binding peptide that is selected to specifically and selectively bind to a target site. The method for forming targeted drug delivery nanocarriers includes the steps of obtaining a plurality of bacteriophage displaying a binding peptide for a desired target site, treating the bacteriophage with a denaturing agent, mixing the treated bacteriophage with a plurality of carrier particles and purifying the mixture to obtain a plurality of targeted drug delivery nanocarriers.

Abstract: The invention is based on the discovery that certain biarsenical molecules react with specified target sequences, thereby providing a facile means for labeling polypeptides containing the target sequence. The invention is useful in creating stable mammalian cell lines expressing a certain tetracysteine tagged polypeptides, thereby overcoming toxicity associated with native tetracysteine. In addition, the invention allows for orthogonal labeling of polypeptides, thereby allowing for the observation of protein-protein interactions and conformational changes in proteins, for example.

Abstract: Recombinant adenoviruses or pseudoviruses having a deletion of all or part of the region of the adenovirus genome corresponding to nucleotides 311 to 499 in canine adenovirus type 2 (GeneBank J04368). Therapeutic methods of using the recombinant adenovirus or pseudovirus.

Abstract: The inventors discovered for the first time the nucleotide sequence of the human DR5 gene promoter, the nucleotide sequence of the human Siah-1 gene promoter and what appear to be the core promoter regions thereof. The present invention further provides a screening method for substances which regulate promoter activity, comprising a step of bringing a test substance into contact with cells holding a vector which comprises this DNA together with a reporter gene ligated expressibly to this DNA, and a step of detecting changes in the expressed amount of the reporter gene due to contact with the test substance. This screening method is a method of very efficiently selecting anti-cancer drugs and the like.

Abstract: This invention provides a composition comprising the following polynucleotide probes: IL7R (AA485865) (SEQ ID NO:7), NDRGI (AA486403) (SEQ ID NO:8), EST1 (H50345) (SEQ ID NO:9), TRPC1 (AA017132) (SEQ ID NO:10), GFRA1 (AA512935) (SEQ ID NO:11), EST2 (AA454543) (SEQ ID NO:12), CLDN10 (R54559) (SEQ ID NO:13), DNALI1 (R93087) (SEQ ID NO:14), RBP5 (AA453198) (SEQ ID NO:15), EST3 (AA621761) (SEQ ID NO:16), EST4 (N63706) (SEQ ID NO:17), PCOLCE (AA670200) (SEQ ID NO:18), TDO2 (T72398) (SEQ ID NO:19), EST5 (T47454) (SEQ ID NO:20), HIST1H2BD (N33927) (SEQ ID NO:21), PXMP2 (N70714) (SEQ ID NO:22), ACAS2 (AA455146) (SEQ ID NO:23), ANAPC7 (T68445) (SEQ ID NO:24), EST6 (AA576580) (SEQ ID NO:25), RBP5 (N92148) (SEQ ID NO:26), ANXAI (H63077) (SEQ ID NO:27), CKB (AA894557) (SEQ ID NO:28), ITGBL1 (N52533) (SEQ ID NO:29), KPNA2 (AA676460) (SEQ ID NO:30), EST7 (W90740) (SEQ ID NO:31) and MEG3 (W85841) (SEQ ID NO:32).

Abstract: The present invention relates to a transcriptional regulatory sequence with enhanced tumor-specificity and strength and a recombinant vector comprising the transcriptional regulatory sequence. More particularly, the present invention relates to a transcriptional regulatory sequence comprising a human telomere reverse transcriptase (hTERT) promoter linked to a nucleotide sequence that comprises one or more c-Myc binding sites and/or one or more Sp1 binding sites, and a recombinant vector comprising a certain gene that is operably linked to the above transcriptional regulatory sequence.

Abstract: Cells are genetically modified to express a luminophore, e.g., a modified (F64L, S65T, Y66H) Green Fluorescent Protein (GFP, EGFP) coupled to a component of an intracellular signalling pathway such as a transcription factor, a cGMP- or cAMP-dependent protein kinase, a cyclin-, calmodulin- or phospholipid-dependent or mitogen-activated serine/threonin protein kinase, a tyrosine protein kinase, or a protein phosphatase (e.g. PKA, PKC, Erk, Smad, VASP, actin, p38, Jnk1, PKG, IkappaB, CDK2, Grk5, Zap70, p85, protein-tyrosine phosphatase 1C, Stat5, NFAT, NFkappaB, RhoA, PKB). An influence modulates the intracellular signalling pathway in such a way that the luminophore is being redistributed or translocated with the component in living cells in a manner experimentally determined to be correlated to the degree of the influence.

Abstract: The present invention relates to a DNA vaccine consisting of a vector expressing a part or the whole of a polypeptide regulating production of an inflammatory cytokine, a pharmaceutical composition comprising the DNA vaccine, and a method of preventing or treating an inflammatory disease by using the pharmaceutical composition.

Abstract: The present invention provides methods and compositions for generating novel nucleic acid molecules through targeted spliceosomal mediated RNA trans-splicing. The compositions of the invention include pre-trans-splicing molecules (PTMs) designed to interact with a SERPINA1 target precursor messenger RNA molecule (target pre-mRNA) and mediate a trans-splicing reaction resulting in the generation of a novel chimeric RNA molecule (chimeric RNA). In particular, the PTMs of the present invention include those genetically engineered to interact with SERPINA1 target pre-mRNA so as to result in correction of SERPINA1 genetic defects responsible for AAT deficiency. The PTMs of the invention may also comprise sequences that are processed out of the PTM to yield duplex siRNA molecules directed specifically to mutant SERPIN A1 mRNAs. Such duplexed siRNAs are designed to reduce the accumulation of toxic AAT protein in liver cells.

Abstract: A DNA composition effective for inhibiting endothelial cell proliferation comprises a DNA construct operably encoding a vascular endothelial growth factor (VEGF) receptor polypeptide, which can be a full length VEGF receptor protein or an immunogenic fragment thereof. This invention provides DNA compositions that encode VEGF receptor-2 (KDR), VEGF receptor-1 (Flt-1), or Flk-1 (the murine homolog of KDR), as well as methods of using such a DNA composition to inhibit vascular endothelial cell proliferation in the tumor micro-environment. Angiogenesis inhibition and subsequent decrease in tumor growth and dissemination is achieved.

Abstract: Insect cells are stored in a small gas cylinder, and the small gas cylinder is charged with nitrogen gas to pressurize the cylinder. The charged gas is exhausted at once to crush the cells to provide the objective cell extract with translation activity and glycosylation activity. As this method is gentler than the conventional cell-crushing method employing a homogenizer, in addition to translation factors, factors carrying glycosylation activity can also be recovered. As a result, an in-vitro glycoprotein synthesis system capable of performing translation to post-translation glycosylation can be produced.

Abstract: Provided is a method of preparing a proliferation-regulated recombinant adenoviral vector effectively, comprising preparing a proliferation-regulated vector plasmid by preparing a restriction enzyme-recognizing sequence in a vector plasmid having a proliferation-regulating unit and having an E1A region, a protein-coding region in a E1B region, a poly(A) signal sequence, and a recombinase-recognizing sequence in that order from upstream, by deleting an endogenous promoter in the E1A region or an endogenous promoter regulating expression of the protein-coding gene in one protein-coding region of the E1B region thereof and inserting the restriction enzyme-recognizing sequences in the deficient site, and introducing a promoter expressing specifically in a target organ in the restriction enzyme-recognizing sequence in the restriction enzyme-recognizing sequence; and additionally, integrating the proliferation-regulated vector plasmid into a vector plasmid having a adenoviral genome prepared by deleting the E1 regi

Abstract: The present invention relates to the field of glycosylation engineering of proteins. More particularly, the present invention relates to glycosylation engineering to generate proteins with improved therapeutic properties, including antibodies with increased antibody-dependent cellular cytotoxicity.

Abstract: Provided are processes for culturing cells derived from embryonic retinoblast cells immortalized by adenovirus E1 sequences, such as PER.C6® cells, to improve product yields from such cells. Feed strategies for such cells and cultures with very high cell densities are provided, resulting in high yields of products, such as recombinant antibodies.

Abstract: In the present invention are disclosed concatemers of concatenated expression cassettes and vectors that enable the synthesis of such concatemers. The concatemer comprises in the 5??3? direction a cassette of nucleotide sequence of the general formula [rs2-SP-PR-X-TR-SP-rs1]n wherein rs1 and rs2 together denote a functional restriction site, SP individually denotes a spacer of at least two nucleotide bases, PR denotes a promoter, capable of functioning in a cell, X denotes an expressible nucleotide sequence, TR denotes a terminator, and SP individually denotes a spacer of at least two nucleotide bases, and n>/=2, and wherein at least a first cassette is different from a second cassette. The main purpose of these concatemers is the controllable and co-ordinated expression of large numbers of heterologous genes in a single host. Furthermore, the invention relates to a concatemer of cassettes of nucleotide sequences and a method for preparing the concatemers.

Abstract: The invention provides an expression cassette comprising a DNA sequence encoding amino acids 1-99 of human preproenkephalin, a DNA sequence encoding a precursor of a carboxy-amidated peptide flanked by dibasic cleavage sites and optionally a DNA sequence encoding a marker protein (such as Enhanced Green Fluorescent Protein (GFP)) all in operable linkage and under control of a promoter. Where the encoded precursor of a carboxy-amidated peptide is an agonist for an opioid receptor, the invention further provides a method of treating neuropathic pain by administering the gene transfer vector comprising such an expression cassette to a patient.

Abstract: The present invention provides a perfusion solution comprising specific metabolic agents, antioxidant agents, and membrane stabilizer agents that can help improve preservation, organ viability, and in some cases recover organs that would otherwise being unusable for transplantation. In a further embodiment, the perfusion solution can be used in combination with hypothermic machine perfusion. It has been found that combination of the perfusion solution and hypothermic machine perfusion can help prevent or reduce further damage to the organ and restore the organ's anti-oxidant system, stabilize the cellular cytoskeleton and cellular membranes, inhibit arachidonic acid pathway, provide oncotic support, reduce interstitial edema formation, and help restore energy stores within the organ. As a result, the method can be used to improve the viability of otherwise marginal donor organs.

Abstract: Methods and compositions for generating novel nucleic acid molecules through targeted spliceosome mediated RNA trans-splicing that result in expression of a apoAI protein, an apoAI variant, the preferred embodiment referred to herein as the apoAI Milano variant, a pre-pro-apoAI or an analogue of apoAI. The methods and compositions include pre-trans-splicing molecules (PTMs) designed to interact with a target precursor messenger RNA molecule (target pre-mRNA) and mediate a trans-splicing reaction resulting in the generation of a novel chimeric RNA molecule (chimeric RNA) capable of encoding apoAI, the apoAI Milano variant, or an analogue of apoAI. The expression of this apoAI protein results in protection against vascular disorders resulting from plaque build up, i.e., atherosclerosis, strokes and heart attacks.