Abstract: The present invention relates to viral transformation method, particularly foamy virus-mediated transformation method. The present invention relates to the transfer of transgene into cells by the safe and efficient transfer of RNA encoding foamy components. The present invention has therefore therapeutic interest, especially in the field of gene therapy.

Abstract: The presently disclosed subject matter provides compositions and methods for the expression of CRISPR guide RNAs using the H1 promoter. In particular, compositions and methods are provided for the use of the H1 promoter to express CRISPR guide RNA (gRNA) with altered specificity of the 5? nucleotide, as well as use of the H1 promoter sequence as a bidirectional promoter to express Cas9 nuclease and the gRNA simultaneously. Compositions and methods are also provided for the expression and regulation of gRNA expression in vivo through the use of RNA ribozymes and regulatable aptazymes.

Abstract: Provided are polynucleotides containing a modified PRE having a variant X gene that includes one or more stop codons not present in an unmodified, such as wild-type, hepatitis X gene. Also provided are polynucleotides containing a modified PRE having a variant X gene that includes one or more degradation sequences not present in an unmodified, such as wild-type, hepatitis X gene. The modified PRE can be operably linked to a nucleic acid encoding a recombinant protein. Also provided are expression cassettes, viral vectors and cells containing the polynucleotides, and compositions and methods of use thereof.

Abstract: Compounds and pharmaceutical compositions are contemplated that inhibit N-acyl-ethanolamine-hydrolyzing acid amidase (NAAA) to so increase the concentration of the substrate of NAAA, palmitoylethanolamide (PEA). NAAA inhibition is contemplated to be effective to alleviate conditions associated with a reduced concentration of PEA. Among other uses, various NAAA inhibitors are especially contemplated as therapeutic agents in the treatment of inflammatory diseases.

Abstract: The object is to provide an expression vector capable of expressing with a higher expression efficiency a protein derived from a foreign structural gene by genetic engineering using a yeast of the genus Schizosaccharomyces as a host, a cloning vector to produce the expression vector, a method for producing the expression vector, a transformant containing an expression cassette of the expression vector, a method for producing the transformant, and a method for producing a protein using the transformant. A cloning vector comprising a promoter capable of functioning in a yeast of the genus Schizosaccharomyces, a cloning site for introducing a foreign structural gene which is located downstream from the promoter and is regulated by the promoter, and ihc2 gene terminator of the yeast of the genus Schizosaccharomyces, and an expression vector comprising the promoter, the foreign structural gene and the ihc2 gene terminator.

Abstract: Disclosed are nucleic acid, RNA, and protein expression profiles which are indicative of the onset, predisposition to the onset and/or progression of a large intestine neoplasm. More particularly disclosed are nucleic acid molecules, the expression profiles of which are indicative of the onset and/or progression of a colorectal neoplasm, such as an adenoma or an adenocarcinoma. The expression profiles of the present invention are useful in a range of applications including, but not limited to, those relating to the diagnosis and/or monitoring of colorectal neoplasms, such as colorectal adenomas and adenocarcinomas. Further disclosed are methods of screening a subject for the onset, predisposition to the onset and/or progression of a large intestine neoplasm by screening for modulation in the expression profile of the disclosed nucleic acid molecule markers.

Abstract: This disclosure describes techniques for creating stable, targeted mutations in Spirulina (Arthrospira) and Spirulina having stable, targeted mutations.

Abstract: The present disclosure provides compositions and methods useful for tissue engineering including a composition having chemically modified RNA (cmRNA) encapsulated in or complexed with a non-viral delivery vehicle and a biocompatible, bioresorbable scaffold and methods of using the composition to regenerate, for example, bone tissue.

Abstract: Methods for the identification of variant recognition sites for rare cutting engineered double strand break inducing agents and compositions thereof are provided. Further provided are nucleic acid constructs, yeast, plants, plant cells, explants, seeds and grain having the of variant recognition sites. Various methods of identifying variant recognition sites with increased substrate activity for a rare cutting engineered double strand break inducing agents are provided.

Abstract: A composition, for diagnosing the possibility of onset of pancreatic cancer including an agent measuring the expression level of marker protein of diagnosing the possibility of onset of pancreatic cancer, or mRNA expression level of a gene encoding the protein, a kit including the composition, and a method of diagnosing pancreatic cancer using the maker are provided. The diagnostic markers of pancreatic cancer in accordance with the present disclosure are useful for predicting or diagnosing the onset, the possibility of onset, and the severity of pancreatic cancer in an early stage, and are also applied to a study on the tumorigenesis of pancreatic cancer. In addition, the diagnosis method of the present invention allows for the convenient detection of pancreatic cancer in a non-invasive manner in a sample such as blood.

Abstract: Control Devices are disclosed including RNA destabilizing elements (RDE), RNA control devices, and destabilizing elements (DE) combined with Chimeric Antigen Receptors (CARs) or other transgenes in eukaryotic cells. Multicistronic vectors are also disclosed for use in engineering host eukaryotic cells with the CARs and transgenes under the control of the control devices. These control devices can be used to optimize expression of CARs in the eukaryotic cells so that, for example, effector function is optimized. CARs and transgene payloads can also be engineered into eukaryotic cells so that the transgene payload is expressed and delivered after stimulation of the CAR on the eukaryotic cell.

Abstract: Control Devices are disclosed including RNA destabilizing elements (RDE), RNA control devices, and destabilizing elements (DE) combined with Chimeric Antigen Receptors (CARs) or other transgenes in eukaryotic cells. Multicistronic vectors are also disclosed for use in engineering host eukaryotic cells with the CARs and transgenes under the control of the control devices. These control devices can be used to optimize expression of CARs in the eukaryotic cells so that, for example, effector function is optimized. CARs and transgene payloads can also be engineered into eukaryotic cells so that the transgene payload is expressed and delivered after stimulation of the CAR on the eukaryotic cell.

Abstract: The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotide molecules encoding low density lipoprotein receptor comprising at least one mutation (e.g., an LDLR signally enhancing mutation).

Abstract: Provided herein are methods and compositions for analyzing nucleic acids associated with single cells using nucleic acid barcodes. According to some embodiments, a method for producing one or more polynucleotides of interest comprises: obtaining a plurality of RNAs associated with one or more samples, wherein the samples are obtained from one or more subjects, each RNA is associated with a single sample, and the RNAs associated with each sample are present in a separate reaction volume; adding an adapter molecule to the RNAs associated with each sample, wherein the adapter molecule is generated using an enzymatic reaction and comprises a universal priming sequence, a barcode sequence, and a binding site; and incorporating the barcode sequence into one or more polynucleotides associated with each sample, thereby producing the one or more polynucleotides of interest.

Abstract: The present invention relates to polypeptides which are Gal2 variants comprising at least one amino acid substitution at a position corresponding to M435, and optionally further amino acid substitution(s). The present invention further relates to nucleic acid molecules encoding the polypeptides and to host cells containing said nucleic acid molecules. The present invention further relates to a method for the production of bioethanol and/or other bio-based compounds, comprising the expression of said nucleic acid molecules, preferably in said host cells. The present invention also relates to the use of the polypeptides, nucleic acids molecule or host cells for the production of bioethanol and/or other bio-based compounds, and/or for the recombinant fermentation of biomaterial containing pentose(s), preferably D-xylose and/or L-arabinose.

Abstract: The invention provides nucleic acids and nucleic acid expression vectors containing optimized mGluR6 promoters for expression of transgenes in the retina. The compositions and methods of the invention are useful for expression of gene products to preserve, improve, or restore phototransduction or vision.

Abstract: The present invention regards a new and Improved HVT-vectored ND-IBD vaccine, comprising a recombinant HVT comprising the VP2 gene from IBDV and the F gene from NDV to a target animal. The recombinant HVT can be used in a vaccine for poultry, which displayed good viral vector replication, effective expression of the NDV F- and IBDV VP2 genes, improved immunoprotection against ND and IBD, and improved genetic stability over prior art constructs.

Abstract: Compositions are provided that comprise a recombinant vector carrying a nucleic acid sequence encoding a fragment of CEP290 lacking all or part of its N-terminal and C-terminal inhibitory regions, under the control of regulatory sequences which express the product of said gene in a selected cell of a mammalian subject, and a pharmaceutically acceptable carrier. These and other compositions are disclosed with are useful in methods for treating a mammalian subject having a disease associated with a CEP290 mutation, such as Lebers Congenital Amaurosis.

Abstract: The invention relates to the identification and selection of differentially expressed transcripts (biomarker) in tumor cells. Specific determination of the level of these biomarkers can be used for screening and diagnosis of prostate cancer. Clinical application of assays based on these biomarker help reduce the high number of false positives of current standard screening assays.

Abstract: This invention is directed to a vector which comprises a promoter operably linked to a nucleic acid sequence encoding a therapeutic gene that blocks allergic reactions. The invention is also directed to a composition comprising the vector and method of using the vector to reduce or inhibit an immune response or allergic reaction to an allergen in a mammal.