Abstract: Methods and compositions are provided for assessing, treating, and preventing diseases, especially cancer, using cancer-associated targets (“CAT”). Methods and compositions are also provided for determining or predicting the effectiveness of a treatment for these diseases or for selecting a treatment, using CAT. Methods and compositions are further provided for modulating cell function using CAT. Also provided are compositions that modulate CAT (e.g., antagonists or agonists), such as antibodies, proteins, small molecule compounds, and nucleic acid agents (e.g., RNAi and antisense agents), as well as pharmaceutical compositions thereof. Further provided are methods of screening for agents that modulate CAT, and agents identified by these screening methods.

Abstract: The present invention concerns particular G-quartet oligonucleotides that are employed for the treatment and/or prevention of cancer. In specific cases, the G-quartet oligonucleotides inhibit HIF-1?.

Abstract: The invention provides GPR17 modulators, methods of screening and use thereof for diagnosis and therapy of diseases or dysfunctions involving GPR17 activation, particularly ischemic brain damage.

Abstract: An arginine-grafted bioreducible poly(disulfide amine) (“ABP”) as a reagent for efficient and nontoxic gene delivery is described. ABP forms positively charged nano-particles of less than 200 nm with siRNA. ABP is biodegraded under reducing conditions, such as in the cytoplasm. ABP exhibits much higher transfection efficiency than polyethyleneimine in mammalian cells and exhibits no cytotoxicity. ABP is an effective delivery vehicle for gene silencing with siRNA and may be used for treating cancer.

Abstract: An arginine-grafted bioreducible poly(disulfide amine) (“ABP”) as a reagent for efficient and nontoxic gene delivery is described. ABP forms positively charged nano-particles of less than 200 nm with plasmid DNA. ABP is biodegraded under reducing conditions, such as the cytoplasm. ABP exhibits much higher transfection efficiency than polyethyleneimine in mammalian cells and exhibits no cytotoxicity.

Abstract: Antisense oligonucleotide compounds, compositions and methods are provided for down regulating the expression of TLR3. The compositions comprise antisense oligonucleotides targeted to nucleic acids encoding TLR3. The compositions may also comprise antisense oligonucleotides targeted to nucleic acids encoding TLR3 in combination with other therapeutic and/or prophylactic compounds and/or compositions. Methods of using these compounds and compositions for down-regulating TLR3 expression and for prevention or treatment of diseases wherein modulation of TLR3 expression would be beneficial are provided.

Abstract: Restenosis in a subject can be treated by administering to a tissue, e.g., a blood vessel, of the subject an agent that increases SERCA activity. For example, a stent that is coated with the agent can be introduced into a blood vessel.

Abstract: Immunostimulatory polymers that contain certain sequence-dependent immunostimulatory RNA motifs and methods for the use of such immunostimulatory polymers and compositions containing such polymers are provided according to the invention. The sequence-dependent immunostimulatory RNA motifs and the polymers incorporating such motifs are potent and selective inducers of TLR7 and the TLR7-associated cytokine IFN-?.

Abstract: This invention provides methods of regulating fat storage in tissue by modulating the levels of Fibrate Induced Transcript 1 (FIT1) and/or Fibrate Induced Transcript 2 (FIT2), as well as diagnostic screens for disorders and conditions involving regulation of fat storage in tissue, assays for identifying agents that can regulate fat storage in tissue through modulating the levels of FIT1 and/or FIT2, and genetically altered mammals in which expression of FIT1 and/or FIT2 is altered in one or more tissue.

Abstract: In the absence of substantial sequence overlap between a recombinant adenoviral vector and the genome of a packaging cell, helper-dependent E1-containing particles (HDEP) can be formed at low frequency. Provided are means and methods for reducing or preventing the generation of HDEP. To this purpose, novel packaging cells and methods of making these are provided.

Abstract: The present invention relates to double-stranded oligonucleotides, pharmaceutical compositions thereof, and use of such double-stranded oligonucleotides and pharmaceutical compositions to modulate nociceptive signaling in a cell or prevent and/or treat pain in a patient.

Abstract: A medical device for implantation into vessels or luminal structures within the body is provided, which stimulates positive blood vessel remodeling. The medical device, such as a stent and a synthetic graft, is provided with a coating with a pharmaceutical composition containing a controlled-release matrix and one or more pharmaceutical substances for direct delivery of drugs to surrounding tissues. The coating on the medical device further comprises one or more barrier layers, and a ligand such as a peptide, an antibody or a small molecule for capturing progenitor endothelial cells in the blood contacting surface of the device for restoring an endothelium at the site of injury. In particular, the drug-coated stents are for use, for example, in balloon angioplasty procedures for preventing or inhibiting restenosis.

Abstract: The present invention relates to the use of live attenuated bacteria for the manufacture of a vaccine for submucosal administration.

Abstract: The invention provides a method of identifying candidate agents to test for treating heart failure involving diastolic impairment, the method comprising: testing an agent to determine whether it (a) binds to serum response factor (SRF), (b) reduces SRF binding to a serum response element (SRE), or (c) reduces SRF protein levels in a cell; wherein if the agent does one or more of (a), (b), and (c), it is identified as a candidate agent.

Abstract: The present invention describes reagents and methods for using concatermized double-stranded oligonucleotide molecules (CODN) for transcription factor decoys. In one embodiment, the concatemers consist of a variable number of end-to-end copies of a short dsDNA containing a sequence or sequences that act as transcription factor decoys.

Abstract: Methods of treating a tumor in a subject include identifying a subject having, at risk for, or suspected of having a tumor, and administering to the subject an effective amount of an IGFBP7 agent if the tumor has increased Ras-BRAF-MEK-Erk signaling, is dependent for growth and/or survival upon the Ras-BRAF-MEK-Erk signaling pathway, and/or expresses an activated or oncogenic BRAF or RAS.

Abstract: The ACP1 *A allele provides a means for diagnosing susceptibility of a human subject to hyperlipidemia, especially hyperlipidemia associated with metabolic syndrome, a means for treating, or preventing the onset of, hyperlipidemia and metabolic syndrome, and a means for screening and identifying drugs suitable for use in treating or preventing hyperlipidemia, especially hyperlipidemia associated with metabolic syndrome. Diagnostic kits are also provided.

Abstract: The present invention relates to processes for production of Very Long Chain Polyunsaturated Fatty Acids (VLC-PUFAs). The present invention also relates to compositions (e.g., nutritional supplements and food products) containing such VLC-PUFAs. In one embodiment, the present invention is directed to methods for biosynthesis and production of the VLC-PUFAs described herein (particularly C28-C38 PUFAs, also referred to herein as supraenes or supraenoics) by the expression, in a production host cell, of the full or partial sequence(s) of Elovl4 DNA/mRNA nucleic acids or ELOVL4 protein sequences encoded thereby, from any species (prokaryotic or eukaryotic) for use in the biosynthesis, production, purification and utilization of VLC-PUFAs in particular by the elongation of C18-C26 saturated fatty acids and PUFAs.

Abstract: Methods useful in the regulation of myocardial contraction are disclosed. The methods are useful in the regulation of heart function. The invention reveals that sorcin overexpression enhances cardiac contractile performance and establishes the concept of sorcin as a regulator of myocardial contractility. The invention also provides screening assays that allow for the identification of agents that modulate sorcin expression. Such agents are useful, for example, for diagnosing cardiac contractile function associated disorders in subjects, and treating the subjects with the agents identified as being able to modulate sorcin expression.

Abstract: The invention relates to a transgenic mouse model with deficient respiratory chain function in dopamine (DA) neurons. By suppressing or deleting the mitochondrial transcription factor A (Tfam) in DA neurons, a mouse model is obtained, which reproduces key pathophysiological features of Parkinson's disease (PD), i.e., slow progressive loss of DA terminals in striatum and loss of DA neurons in substantia nigra pars compacta; alpha-synuclein immunoreactivity including intracellular inclusions similar to Lewy bodies in affected areas prior to and during cell loss; progressive movement disorder associated with abnormal gait, tremor and rigid limbs. The mouse model can be used to develop pharmacological, gene therapy or cell therapy treatments for PD.