Abstract: Small interfering RNAs (siRNAs) or small hairpin RNA (shRNAs) and compositions comprising same are provided that target human cyclophilin A (CyPA) to inhibit Hepatitis C (HCV) infection. Such siRNA and shRNAs may have a length of from about 19 to about 29 contiguous nucleotides corresponding to a specific region of human cyclophilin A (CyPA) cDNA of from about nucleotide 155 to about nucleotide 183 having particular potency against CyPA and HCV. Such siRNA and shRNAs may be formulated as naked compositions or pharmaceutical compositions. DNA polynucleotides, plasmids, and viral or non-viral vectors are also provided that encode siRNA or shRNA molecules, which may be delivered directly to cells or in combination with delivery agents, such as lipids, polymers, encapsulated lipid particles, such as liposomes. Methods for treating, managing inhibiting, preventing, etc., HCV infection using such siRNA and shRNAs and compositions comprising same are also provided.

Abstract: Methods and compositions for the treatment of pathologic conditions of the central nervous system (CNS) by means of intranasal administration of a composition that modulates, by means of RNA interference, the expression and/or activity of genes involved in above-mentioned conditions.

Abstract: The invention provides for systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are vectors and vector systems, some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells and methods for selecting specific cells by introducing precise mutations utilizing the CRISPR/Cas system.

Abstract: A novel transformation system in the field of filamentous fungal hosts for expressing and secreting heterologous proteins or polypeptides is described. The invention also covers a process for producing large amounts of polypeptide or protein in an economical manner. The system comprises a transformed or transfected fungal strain of the genus Chrysosporium, more particularly of Chrysosporium lucknowense and mutants or derivatives thereof. It also covers transformants containing Chrysosporium coding sequences, as well expression-regulating sequences of Chrysosporium genes. Also provided are novel fungal enzymes and their encoding sequences and expression-regulating sequences.

Abstract: The invention provides a conjugate comprising (i) a nucleic acid which is complementary to a target nucleic acid sequence and which expression prevents or reduces expression of the target nucleic acid and (ii) a selectivity agent which is capable of binding with high affinity to a receptor which can be internalised by the cell in response to the binding of said selectivity agent. The conjugates of the present invention are useful for the delivery of the nucleic acid to a cell of interest and thus, for the treatment of diseases which require a down-regulation of the protein encoded by the target nucleic acid as well as for the delivery of contrast agents to the cells for diagnostic purposes.

Abstract: Isolated polynucleotides comprising a CCKBR mini-promoters are provided. The mini-promoter may be operably linked to an expressible sequence, e.g. reporter genes, genes encoding a polypeptide of interest, regulatory RNA sequences such as miRNA, siRNA, anti-sense RNA, etc., and the like. In some embodiments a cell comprising a stable integrant of an expression vector is provided, which may be integrated in the genome of the cell. The promoter may also be provided in a vector, for example in combination with an expressible sequence. The polynucleotides find use in a method of expressing a sequence of interest, e.g. for identifying or labeling cells, monitoring or tracking the expression of cells, gene therapy, etc.

Abstract: Provided herein are methods, compounds, and compositions for reducing expression of GCGR mRNA and protein in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate metabolic disease, for example, diabetes, or a symptom thereof.

Abstract: Disclosed herein are compositions, systems and methods for delivery of proteins of interest using adeno-associated virus (AAV) vectors.

Abstract: The invention relates to a DNA consisting of the nucleotide sequence shown in SEQ ID NO: 1, or a nucleotide sequence the same or substantially the same as a partial nucleotide sequence thereof containing at least one HRE consensus sequence, which transiently has a transcription promoting activity in a vascular endothelial cell-specific manner in a hypoxic state, or a vector containing a promoter containing the DNA, and the like. The prophylaxis and/or treatment, as well as diagnosis, of acute ischemic diseases are enabled by connecting a prophylactic and/or therapeutic gene or a reporter gene to the downstream of the vector and administering same to mammals.

Abstract: Antisense compounds, compositions and methods are provided for modulating the expression of kinesin-like 1. The compositions comprise antisense compounds, particularly antisense oligonucleotides, targeted to nucleic acids encoding kinesin-like 1. Methods of using these compounds for modulation of kinesin-like 1 expression and for treatment of diseases associated with expression of kinesin-like 1 are provided.

Abstract: The invention provides for engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are compositions and methods related to components of a CRISPR complex particularly comprising a Cas ortholog enzyme.

Abstract: Provided are antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon skipping, and methods of use thereof to treat muscular dystrophy.

Abstract: An isolated polynucleotide functional as a promoter in eukaryotic cells is disclosed. The isolated polynucleotide includes an endothelial specific enhancer element as detailed herein. Further disclosed is a method of expressing a nucleic acid sequence of interest in endothelial cells.

Abstract: Compounds, compositions and methods are provided for modulating the expression of C-reactive protein. The compositions comprise oligonucleotides, targeted to nucleic acid encoding C-reactive protein. Methods of using these compounds for modulation of C-reactive protein expression and for diagnosis and treatment of disease associated with expression of C-reactive protein are provided.

Abstract: The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Tumor Necrosis Factor Receptor 2 (TNFR2), in particular, by targeting natural antisense polynucleotides of Tumor Necrosis Factor Receptor 2 (TNFR2). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of TNFR2.

Abstract: The present invention provides non-coding regulatory element polynucleotide molecules isolated from the lipid transfer protein (LTP) gene of Oryza sativa and useful for expressing transgenes in plants. The invention further discloses compositions, polynucleotide constructs, transformed host cells, transgenic plants and seeds containing the Oryza sativa regulatory polynucleotide sequences, and methods for preparing and using the same.

Abstract: A method of mRNA production for use in transfection is provided, that involves in vitro transcription of PCR generated templates. This RNA can efficiently transfect different kinds of cells. This approach results in increased efficiency (fidelity and productivity) of mRNA synthesis and is less time consuming because it does not require cloning, and also consequently eliminates the unwanted errors and effects related to RNA made on DNA templates obtained with cloning techniques. The results of transfection of RNAs demonstrate that RNA transfection can be very effective in cells that are exceedingly difficult to transfect efficiently with DNA constructs. The method can be used to deliver genes into cells not- or only poorly transfectable for DNA, in vitro and in vivo.

Abstract: An isolated polynucleotide functional as a promoter in eukaryotic cells is disclosed. The isolated polynucleotide includes an endothelial specific enhancer element as detailed herein. Further disclosed is a method of expressing a nucleic acid sequence of interest in endothelial cells.

Abstract: Compositions for modulating the expression of a protein in a target cell comprising at least one RNA molecule which comprises at least one modification conferring stability to the RNA, as well as related methods, are disclosed.

Abstract: Presently provided are a cineraria-derived polynucleotide which can act as a promoter for a coding region of flavonoid 3?,5?-hydroxylase (F3?5?H and a F3?5?H gene construct which carries the polynucleotide. The cineraria-derived petal-specific promoter can be as polynucleotide having the nucleotide sequence depicted in SEQ ID NO: 9 or a polynucleotide which can hybridize with the polynucleotide having the nucleotide sequence depicted in SEQ ID NO: 9 under stringent conditions and can act as a promoter for a coding region of flavonoid. 3?,5?-hydroxylase (F3?5?H). The gene construct can be used, for inhibiting the expression of a gene in an RNAi method, which has, as a loop, a polynucleotide comprising the whole or a part of a first intron of the presently provided cineraria-derived polynucleotide.

Abstract: The present invention is concerned with means and methods for allowing tissue specific and, in particular, seed specific expression of genes. The present invention, accordingly, relates to a polynucleotide comprising an expression control sequence which allows seed specific expression of a nucleic acid of interest being operatively linked thereto. Moreover, the present invention contemplates vectors, host cells, non-human transgenic organisms comprising the aforementioned polynucleotide as well as methods and uses of such a polynucleotide.

Abstract: The present invention relates to a nucleic acid molecule containing a sequence of tricyclo nucleosides joined by internucleoside phosphorothioate linkage. The invention also relates to synthetic antisense oligonucleotides and to methods employing the same.

Abstract: A cyanobacterial host cell, Cyanobacterium sp., that harbors at least one recombinant gene for the production of a chemical compounds is provided, as well as vectors derived from an endogenous plasmid isolated from the cell.

Abstract: The present invention provides novel promoters for use in plants. Specifically, the present invention provides novel chimeric promoters comprising combinations of plant enhancer elements and plant promoters. The present invention also provides DNA constructs; transgenic cells, plants, and seeds containing these novel chimeric promoters; and methods for preparing and using the same.

Abstract: The invention provides several promoters isolated from Oryza sativa, which promoters are capable of driving and/or regulating the expression of an operably linked nucleic acid in a plant. The expression patterns of the promoters according to the invention have been studied in Oryza sativa and some of the promoters displayed specific activity in particular cells, tissues or organs of the plant, while others displayed constitutive expression throughout substantially the whole plant. Some promoters showed weak expression, while others were strongly active.

Abstract: The invention provides several promoters isolated from Oryza sativa, which promoters are capable of driving and/or regulating the expression of an operably linked nucleic acid in a plant. The expression patterns of the promoters according to the invention have been studied in Oryza sativa and some of the promoters displayed specific activity in particular cells, tissues or organs of the plant, while others displayed constitutive expression throughout substantially the whole plant. Some promoters showed weak expression, while others were strongly active.

Abstract: An isolated polynucleotide functional as a promoter in eukaryotic cells is disclosed. The isolated polynucleotide includes an endothelial specific enhancer element as detailed herein. Further disclosed is a method of expressing a nucleic acid sequence of interest in endothelial cells.

Abstract: Lentiviral vectors modified at the 5? LTR or both the 5? and 3? LTR's are useful in the production of recombinant lentivirus vectors. Such vectors can be produced in the absence of a functional tat gene. Multiple transformation of the host cell with the vector carrying the transgene enhances virus production.

Abstract: Provided is a novel high-expression promoter, namely a GAL1 promoter, derived from Kluyveromyces marxianus. Also provided are the following, characterized by the use of the provided high-expression promoter; a recombinant polynucleotide containing said high-expression promoter; a vector containing said recombinant polynucleotide; a transformant obtained by introducing said recombinant polynucleotide or vector into yeast; a method using said transformant for high expression of a target gene; and a method using said transformant to manufacture the gene product of a target gene.

Abstract: Identification of new enhancer sequence has significant utility in the plant functional genomics. The sugarcane bacilliform badnavirus (SCBV) transcriptional enhancer has been identified. This enhancer can be used to increase the rate of transcription from gene promoters and in activation tagging experiments. A ten-fold increase in transcription was observed when a 4× array of the SCBV enhancer was placed upstream of a truncated form of the maize alcohol dehydrogenase minimal promoter. Methods of using the SCBV transcriptional enhancer are described, as are chimeric transcription regulatory regions, constructs, cells, tissues, and organisms that comprise one or more copies of the enhancer.

Abstract: The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of an Antiviral gene, in particular, by targeting natural antisense polynucleotides of an Antiviral gene. The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of Antiviral genes.

Abstract: The present invention relates to the use of regulatory sequences for mediating specific, early transient expression in proliverative neuronal determined cells. Furthermore, the uses of recombinant nucleic acid molecules comprising said defined regulatory sequences for mediating specific, early transient expression in proliverative neuronal determined cells as well as for the generation of non-human transgenic organisms and/or host cells are disclosed. In addition, the invention provides for transgenic non-human animals and/or host cells comprising said regulatory sequences and/or recombinant nucleic acid molecules. The invention also describes methods for the preparation of such vectors, host cells and transgenic non-human animals as well as methods for the detection and/or isolation of neuronal determined cells.

Abstract: The present invention relates to polynucleotides comprising a first nucleic acid sequence for a chromatin element, which is capable of enhancing expression, and at least one second nucleic acid sequence comprising a curved origin motif. Furthermore, the invention relates to a host cell, a non-human transgenic organism, a vector and a kit comprising the aforementioned polynucleotide. Moreover, the invention relates to methods for expressing a polynucleotide of interest.

Abstract: The present invention is directed to promoters of flax conlinin and ?-3 desaturase genes. The promoters guide high levels of the expression exclusively in flax developing seeds. This specific expression pattern concomitant with the biosynthesis of storage lipids and proteins make these promoters particularly useful for seed-specific modification of fatty acid and protein compositions in plant seeds.

Abstract: The present invention concerns Zdhhc2, a new target involved in adipogenesis modulation. Using a siRNA approach, the inventors demonstrated that decrease in Zdhhc2 activity in adipose tissue induces a decrease in adipogenesis. Thus, the present invention relates to modulators of Zdhhc2 activity as well as screening test for identification of modulators of the activity of this target, and their use, especially in pharmaceutical composition, to modulate adipogenesis and thus treat obesity and related disorders.

Abstract: The present disclosure provides a DNA molecule capable of replication in Mycobacteria having a nucleic acid sequence as disclosed in SEQ ID NO: 1, a shuttle vector constructed using it and a transformed cells containing the present vector. The vector of about 18 kb of the present disclosure contains 16 ORFs, a replication origin and a rep-like protein essential for replication. Therefore, the plasmid of the present disclosure can be utilized as a gene delivery system/research, and also in a therapeutic system such as immune therapeutics by effectively delivering proteins or heterologous DNA and expressing the encoded DNA in cells.

Abstract: The present invention relates to isolated rice LP2 promoter sequences and uses thereof.

Abstract: A relationship between cancer and ribonucleic acid (RNA) regulation is described by determining intracellular levels of niRN A regulators. Generally, mRNA levels are decreased in cancer cells that may be a reflection of either reduced mRNA expression and/or increased mRNA degradation. miRNAs are identified that hybridize to an mRNA that are suspected to mediate intracellular mRNA steady state levels. Alternatively, ribonucleic acid binding protein (RBP) levels may also mediate intracellular mRNA steady state levels. In particular, this invention demonstrates an effective clinical management strategy for uterine cell cancers may be implemented by taking advantage of an exemplary relationship between P2X7 mRNA and miRNAs including, but not limited to, miR-186 and/or miR-150.

Abstract: The present invention provides compositions and methods for regulating expression of heterologous nucleotide sequences in a plant. Compositions include a novel nucleotide sequence for a promoter for the gene encoding Sorghum bicolor TIP2-3. A method for expressing a heterologous nucleotide sequence in a plant using the promoter sequences disclosed herein is provided. The method comprises transforming a plant or plant cell with a nucleotide sequence operably linked to one of the promoters of the present invention.

Abstract: Decoy nucleic acid sequences comprising a binding site for a target transcription factor, wherein the binding site is not operably linked to a gene, and wherein the transcription factor comprises a regulator of expression of a gene or genes in Clostridium difficile encoding one or more of: (i) a cellular growth factor; (ii) a cellular toxin; (iii) a cellular sporulation factor; (iv) a cellular stress response; and/or (v) a cellular essential gene are described. Uses of the decoys in antibacterial complexes for the treatment of bacterial infections are also described.

Abstract: The invention provides recombinant DNA molecules and constructs, and their nucleotide sequences, useful for modulating gene expression in plants. The invention also provides transgenic plants, plant cells, plant parts, and seeds comprising a recombinant DNA molecule comprising a DNA molecule operably linked to a heterologous transcribable DNA molecule, as well as methods of their use.

Abstract: The present invention provides a method for diagnosing and determining prognosis of gastric cancer in a subject by detecting suppressed expression of the ZNF545 gene, which in some cases is due to elevated methylation level in the genomic sequence of this gene. A kit and device useful for such a method are also provided.

Abstract: The present invention provides compositions and methods for regulating expression of heterologous nucleotide sequences in a plant. Compositions include a novel nucleotide sequence for a promoter for the gene encoding Sorghum bicolor pLTP. A method for expressing a heterologous nucleotide sequence in a plant using the promoter sequences disclosed herein is provided. The method comprises transforming a plant or plant cell with a nucleotide sequence operably linked to one of the promoters of the present invention.

Abstract: The invention relates to an isolated nucleic acid sequence comprising a promoter, which is a native sequence of Pichia pastoris comprising the nucleic acid sequence of pCS1 of SEQ ID NO:1, or a functionally active variant thereof which is a size variant, a mutant or hybrid of SEQ ID NO:1, or a combination thereof, expression constructs and recombinant host cells comprising the promoter, and a method of producing a protein of interest under the control of the promoter. It further relates to a method to identify a constitutive promoter from eukaryotic cells, and an isolated nucleic acid sequence comprising a promoter which when operatively linked to a nucleotide sequence encoding a protein of interest directs the expression thereof in a host cell at an expression level that is higher than under control of the native pGAP promoter at high and low growth rates.

Abstract: The present invention provides, among other things, oligonucleotide modulators of human 5?-HT2C receptor (HTR2C) and improved methods and composition for treating HTR2C-related diseases, disorders or conditions based on such modulators. In particular, oligonucleotides modulators according to the invention target specific regions in the Exon V/Intron V junction of the human HTR2C pre-mRNA and drive expression of HTR2C Vb splice isoform, leading to increased generation of non-edited strong HTR2C receptor and enhanced serotonin receptor activity.

Abstract: The subject invention concerns materials and methods for providing for cone cell specific expression of a polynucleotide in a human or animal. One aspect of the invention concerns a polynucleotide promoter sequence that directs expression of an operably linked polynucleotide in cone cells. In one embodiment, a polynucleotide of the invention comprises a nucleotide sequence of an interphotoreceptor retinoid-binding protein (IRBP) gene that is positioned upstream of a promoter nucleotide sequence of a cone transducin alpha-subunit (GNAT2) gene. Another aspect of the subject invention concerns methods for expressing a selected polynucleotide in cone cells. The selected polynucleotide can be provided in a polynucleotide of the invention wherein the selected polynucleotide is operably linked to a polynucleotide promoter sequence of the invention. In one embodiment, the selected polynucleotide sequence is provided in a polynucleotide vector of the invention.

Abstract: The present invention provides a trans-splicing ribozyme comprising i) a targeting nucleotide sequence that is complementary to a target nucleotide sequence within a mRNA that is expressed in a cell; contiguous with ii) a catalytic RNA sequence; contiguous with iii) a donor transcript, which donor transcript comprises at least a nucleotide sequence that encodes a trans-activator, wherein when the trans-splicing ribozyme is expressed in a cell, the catalytic RNA sequence cleaves the mRNA and ligates the donor transcript to the mRNA to generate a spliced mRNA which comprises the donor transcript, such that the donor transcript is translated as part of the spliced mRNA in the cell, as well as methods of using the trans-splicing ribozyme. The present invention also provides variants of Cre and other recombinases, as well as method of using the variants.

Abstract: The present invention provides synthetic 5?UTRs comprising a first polynucleotide fragment and a second polynucleotide fragment, wherein the first polynucleotide fragment comprises at least one splice site of a first eukaryotic gene, the second polynucleotide fragment comprises at least a portion of 5? untranslated region of a second eukaryotic gene, and the first polynucleotide fragment is located 5? of the second polynucleotide fragment. In one embodiment, the first polynucleotide fragment comprises the second intron of a sarcoplasmic/endoplasmic reticulum calcium ATPase gene and the second polynucleotide fragment comprises at least a portion of the 5? untranslated region (5?UTR) of a eukaryotic casein gene. The synthetic 5?UTRs are useful for increasing the expression of a transgene when positioned between a promoter and a transgene within an expression vector.

Abstract: The present invention relates to materials and methods for the expression of a gene of interest specifically in seeds of plants, even more specifically in oilseed plants. In particular, the invention provides an expression cassette for regulating seed-specific expression in plants.

Abstract: Isolated polynucleotides comprising a FEV mini-promoters are provided. The mini-promoter may be operably linked to an expressible sequence, e.g. reporter genes, genes encoding a polypeptide of interest, regulatory RNA sequences such as miRNA, siRNA, anti-sense RNA, etc., and the like. In some embodiments a cell comprising a stable integrant of an expression vector is provided, which may be integrated in the genome of the cell. The promoter may also be provided in a vector, for example in combination with an expressible sequence. The polynucleotides find use in a method of expressing a sequence of interest, e.g. for identifying or labeling cells, monitoring or tracking the expression of cells, gene therapy, etc.