Abstract: Disclosed herein are recombinant adenoviruses with one or more nucleotide sequences inserted between two viral transcription units, formulations comprising the recombinant adenoviruses, and methods of treatment using the recombinant adenoviruses. In some embodiments, the one or more nucleotide sequences are inserted in an IX-E2 insertion site and/or an L5-E4 insertion site.

Abstract: The invention provides a polynucleotide sequence (e.g., a gene, e.g., DNA or RNA) encoding UGT1A1 (e.g., expressing human UGT1A1). The invention further provides a vector, such as an adeno-associated virus (AAV) vector (e.g., AAV8) having a vector genome including inverted terminal repeat sequences and a UGT1A1 coding sequence operably linked to one or more expression control sequences (e.g., expression control sequences including a liver-specific promoter). Also provided are compositions containing these AAV vectors and methods of treating Crigler-Nijjar syndrome type I, Crigler-Nijjar syndrome type II, and Gilbert syndrome.

Abstract: A method of preparing a library of small interfering RNA (siRNA) expression systems for producing siRNA for silencing of target genes by inducing degradation of target gene RNA expression products includes: (i) isolating RNA of one or more target genes from a cell population; (ii) generating RNA fragments from the isolated RNA; (iii) converting the RNA fragments into dsDNA fragments; and (iv) cloning the dsDNA fragments into vectors for forming cloned vectors, each vector including one or more promoters and at least one restriction enzyme site capable of accepting the insertion of at least one dsDNA fragment such that siRNA can be produced. Methods for producing siRNA from the siRNA expression system and methods of identifying a functional target gene for treatment by using the siRNA produced from the siRNA expression system and for identifying RNAi therapeutics are also provided.

Abstract: Described herein are CRISPR/Cas9 constructs designed for the C-terminal truncation of human amyloid precursor protein (APP) as well as methods of making and using such a construct. A Cas9 nuclease/gRNA ribonucleoprotein directs cleavage of an APP gene to provide a C-terminal truncated APP having a length of 659, 670, 676, or 686 amino acids, relative to the human or mouse APP sequence.

Abstract: Methods and compositions for generating immune responses using adenovirus vectors that allow multiple vaccinations or in combination with other therapy and vaccinations in individuals with preexisting immunity to adenovirus are provided.

Abstract: The invention relates to an in vitro cell-free expression system incorporating a novel inorganic polyphosphate-based energy regeneration system. In certain embodiments, the invention includes a cell-free expression system where the cellular energy source, ATP, is regenerated from inorganic polyphosphate using a dual enzyme system. In this embodiment, this dual enzyme system may include thermostable Adenosyl Kinase, and/or Polyphosphate Kinase enzymes.

Abstract: Yeast having modified chromosomes are provided. The chromosomes are modified such that at least one of yeast histones H3, H4, H2A or H2B are fully or partially replaced by their human histone counterparts H3, H4, H2A or H2B, respectively. Histone amino acid substitutions are included. Cell fusions with the yeast having the modified chromosomes and non-yeast cells are provided. Methods for screening test agents using the yeast are also provided. Yeast with a mutated yeast DAD1 gene, the mutated DAD1 gene encoding an E50D mutation in yeast DAD1 protein, are provided, and provide a useful genetic background for making the yeast with partially or fully replaced histone(s).

Abstract: The present invention is directed to a method of manufacturing a polyepitopic protein comprising the steps of cloning a blunt-ended DNA sequence by encoding the epitope that is to be cloned into a DNA vector recognized by the endonuclease SmaI or the endonuclease SapI and isolating the polyepitopic protein by transforming a bacterial host cell with such vector.

Abstract: The present invention relates to methods and compositions for determination of and uses of specific methylation patterns indicative of adenoma and carcinoma. In particular, the invention relates to analysis of defined CpG loci that are coordinately methylated in DNAs from cancer and adenoma samples, methods for identifying coordinately methylated loci, and methods of using analysis of coordinately methylated loci in one or more marker regions in the design of assays for adenoma and cancer.

Abstract: A shuttle vector is provided which can be manipulated in various kinds of host cells, thereby providing a novel tool for the field of genetic engineering. Also provided are a prokaryotic host cell and a kit including said shuttle vector, so as to construct expression vectors which contain the target gene using the shuttle vector, thereby producing proteins in various host cells with one single vector.

Abstract: Provided are methods of treating breast cancer in a subject in need thereof comprising administering to the subject an effective amount of an inhibitor of miR-105 or an inhibitor of miR-122 are provided. Also provided herein are methods of determining a level of miR-105 or a level of miR-122 in a subject that has or is at risk for developing breast cancer. The method includes obtaining a biological sample from the subject and determining a level of miR-105 or a level of miR-122 or a combination thereof in the biological sample, wherein a higher level of miR-105 or miR-122 as compared to a control indicates that the subject has or is at risk of developing breast cancer.

Abstract: The present invention is directed to methods and compositions for genome editing and DNA targeting of proteins.

Abstract: Described is a simple plasma-based mammalian expression system that uses the RNA dependent RNA polymerase (RDRP) enzyme systems' activity for expression of recombinant proteins or RNA from viral minigenomes and rescue of recombinant viruses from cDNAs encoding entire genome(s) of negative stranded RNA viruses for use in synthesizing recombinant viruses and developing vaccines. This system will be used for expression of recombinant proteins, therapeutic RNA molecules including anti-sense and/or selecting interfering RNA and Ribozymes. This system can also be used for gene therapy and producing recombinant viruses for production of new vaccines.

Abstract: This invention relates, e.g., to a method for determining if a thyroid tumor in a subject is malignant, comprising determining in a sample from the subject the amount of TERT (telomerase reverse transcriptase) mRNA which lacks the ? sequence and the amount of TERT mRNA in the sample which comprises the ? sequence, wherein a preponderance (e.g., at least about 55%) of TERT mRNA in the sample which comprises the ? sequence indicates that the tumor is malignant, and wherein a preponderance of TERT mRNA which lacks the ? sequence indicates that the tumor is not malignant.

Abstract: The disclosure provides compositions and methods for purification of proteins, including multi-subunit complexes and membrane proteins. Modified colicin-DNAse domains and modified colicin immunity proteins are provided for use in purification methods. Also provided are related nucleic acids, vectors, genetically modified cells and affinity matrices.

Abstract: The invention generally relates to performing sandwich assays in droplets. In certain embodiments, the invention provides methods for detecting a target analyte that involve forming a compartmentalized portion of fluid including a portion of a sample suspected of containing a target analyte and a sample identifier, a first binding agent having a target identifier, and a second binding agent specific to the target analyte under conditions that produce a complex of the first and second binding agents with the target analyte, separating the complexes, and detecting the complexes, thereby detecting the target analyte.

Abstract: Disclosed are whole-cell catalysts, methods of making the whole-cell catalysts, and methods of using the whole-cell catalysts to produce steviol glycosides.

Abstract: The present invention describes host cells for reliable, high yield recombinant protein production, including unstable proteins. The present host cell (e.g., a bacterial cell) is deficient in at least one protease (or a subunit of a protease) such as Clp or ClpP. The host cell may also contain an expression vector that encodes a protein or polypeptide for overexpression.

Abstract: The disclosed Hi-C protocol can identify genomic loci that are spatially co-located in vivo. These spatial co-locations may include, but are not limited to, intrachromosomal interactions and/or interchromosomal interactions. Hi-C techniques may be applied to many different scales of interest. For example, on a large scale, Hi-C techniques can be used to identify long-range interactions between distant genomic loci.

Abstract: Herein is reported a method for the recombinant production of a polypeptide in a eukaryotic cell comprising the steps of (i) cultivating a eukaryotic cell comprising a nucleic acid encoding the polypeptide in a cultivation medium comprising a compound selected from the group consisting of trans-2-methyl 2-pentenoic acid, the broad-spectrum HDAC inhibitor Quisinostat, and the subtype-specific HDAC inhibitor Romidepsin, and (ii) recovering the polypeptide from the cell or the cultivation medium and thereby producing the polypeptide in a eukaryotic cell.