Abstract: Systems and methods to modulate the function of mucosal-associated invariant T (MAIT) cells in the absence of a T cell receptor (TCR) signal are described. The systems and methods can be used to elucidate the function of MAIT cells to assess potential therapeutic strategies for conditions associated with inflammation.

Abstract: The present invention provides new polynucleotide sequences, adeno-associated virus-derived vectors and pharmaceutical compositions containing the same for the treatment of lysosomal storage disorders and specially, for the treatment of mucopolysaccharidosis type IVA or Morquio A syndrome.

Abstract: Described are compositions and methods relating to yeast cells having a genetic mutation that give rise to increased alcohol production, particularly under high dissolved solids growth conditions. Such yeast is well-suited for use in alcohol production to reduce fermentation time and/or increase yields.

Abstract: One aspect of the present disclosure relates to an isolated mutant human estrogen receptor alpha (hER?) that may be used in methods of drug discovery. The isolated mutant hER? can include a DNA-binding domain (DBD), a ligand-binding domain (LBD), and an interface between the DBD and the LBD, wherein at least one tryptophan residue is mutated to a phenylalanine residue.

Abstract: The presently disclosed subject matter relates to targeted integration (TI) host cells suitable for the expression of recombinant proteins, as well as methods of producing and using said TI host cells.

Abstract: The present disclosure provides a DNA-targeting RNA that comprises a targeting sequence and, together with a modifying polypeptide, provides for site-specific modification of a target DNA and/or a polypeptide associated with the target DNA. The present disclosure further provides site-specific modifying polypeptides. The present disclosure further provides methods of site-specific modification of a target DNA and/or a polypeptide associated with the target DNA The present disclosure provides methods of modulating transcription of a target nucleic acid in a target cell, generally involving contacting the target nucleic acid with an enzymatically inactive Cas9 polypeptide and a DNA-targeting RNA. Kits and compositions for carrying out the methods are also provided. The present disclosure provides genetically modified cells that produce Cas9; and Cas9 transgenic non-human multicellular organisms.

Abstract: The present strains and methods of the disclosure relate to genetic modifications in filamentous fungi that give rise to variant strains of filamentous fungi comprising enhanced protein productivity phenotypes. More specifically, as presented, described and exemplified herein, such variant strains of filamentous fungi comprising enhanced protein productivity phenotypes are well-suited for growth in submerged cultures, such as in large-scale production of proteins of interest for commercial applications.

Abstract: The invention relates to peptide linkers and fusion proteins comprising linkers designed for optimizing the activity of the proteins comprised therein, and methods for using the same. The invention further relates to newly designed Cas12a-based adenine base editors.

Abstract: Provided herein are compositions and methods of integrating one or more exogenous donor nucleic acids into one or more exogenous landing pads engineered into a host cell's genome. In certain embodiments, the exogenous landing pads and exogenous donor nucleic acids comprise standardized, compatible homology regions so that exogenous donor nucleic acids can integrate into any of the landing pads, independent of the genomic sequences surrounding the landing pads. In certain embodiments, the methods comprise contacting the host cell comprising landing pads with one or more exogenous donor nucleic acids, and a nuclease capable of causing a double-strand break within the landing pads, and recovering a host cell comprising one or more exogenous donor nucleic acids integrated in any of the landing pads.

Abstract: Methods, reporter gene constructs, and kits for using prostate-specific membrane antigen (PSMA) as an imaging reporter to image a variety of cells and tissues are provided.

Abstract: A method for establishing a biological model on joint toxicity of Caenorhabditis elegans and an application thereof are provided. The method for establishing the biological model includes adding the L1-stage Caenorhabditis elegans to a mixed system of working solution of mycotoxins containing tenuazonic acid and penicillin, K-medium solution and E. coli OP50 to obtain L1-stage Caenorhabditis elegans. The biological model indicates that TeA and PAT have a synergistic effect on the growth, development and reproductive ability of Caenorhabditis elegans, and their toxicity mechanism is related to inducing nematodes and stimulating transcriptional factors of Daf-16 genes. The biological model and detection method are simple to operate and its test period is short.

Abstract: The present disclosure relates to methods of and systems for modifying the transcriptional regulation of stem or progenitor cells to promote their differentiation or reprogramming of somatic cells. Further, the labeling and editing of human genomic loci in live cells with three orthogonal CRISPR/Cas9 components allow multicolor detection of genomic loci with high spatial resolution, which provides an avenue for barcoding elements of the human genome in the living state.

Abstract: A lentiviral vector and a method for delivering an exogenous RNA by the lentiviral vector are provided. The lentiviral vector is prepared by transfecting plasmids containing a genome sequence of the lentiviral vector into a virus-producing cell, collecting a supernatant and concentration. Specifically, according to the principle of combining an RNA-binding protein with an RNA sequence identified by the RNA-binding protein, the RNA-binding protein is integrated into a skeleton of a lentivirus GagPol long-chain protein, and the RNA sequence identified by the RNA-binding protein is connected to the exogenous target RNA, so that the exogenous target RNA is packaged into lentiviral particles during the assembly of the lentiviral particles. The exogenous target RNA can be mRNA, gRNA or RNA with other functions. The present invention can be used in the fields of gene editing, gene therapy, cell therapy, immunotherapy, regenerative medicine and basic biology.

Abstract: The present disclosure relates to compositions of matter and methods used to activate effector nucleic acids and effector targets in vivo via a CRISPR-based cascade system. The compositions and methods achieve non-specific delivery of cascade system components to cells yet the cascade system works in a cell-specific manner.

Abstract: The present disclosure relates to high-throughput detection of polyketides using genetically encoded biosensors.

Abstract: The present invention provides, among other things, methods of quantitating mRNA capping efficiency, particularly for mRNA synthesized in vitro. In some embodiments, the methods comprise chromatographic methods of quantifying capping efficiency and methylation status of the caps.

Abstract: The present invention relates to: acid-resistant yeast to which lactic acid productivity is imparted, and in which the conversion of pyruvate into acetaldehyde is inhibited and, consequently, the ethanol production pathway is inhibited; and a method for producing lactic acid by using same.

Abstract: The present disclosure provides methods of treating subjects having increased serum glucose level and/or hyperglycemia, methods of identifying subjects having an increased risk of developing increased serum glucose level and/or hyperglycemia, and methods of detecting human Solute Carrier Family 39 Member 5 variant nucleic acid molecules and variant polypeptides.

Abstract: The present disclosure provides, inter alia, meiotic drive genes derived from chromosome 3 of the fission yeasts Schizosaccharomyces kambucha and S. pombe and orthologs thereof. In certain embodiments, compositions, methods, and kits are provided for biasing organisms to express such genes and, optionally, to co-express one or more genes of interest in such organisms.

Abstract: A polynucleotide encoding a ThermoCas9 protein from Geobacillus thermodenitrificans and a constitutive promoter are used to engineer eukaryotic cells, e.g. fungi, yeast or algae, so that the ThermoCas9 endonuclease is integrated and expressed from the genome of the cell. Then, a second expression plasmid is used to transfect these ThermoCas9 expressing cells, the second plasmid containing an inducible promoter and a polynucleotide encoding a guide RNA. The guide RNA combines with the ThermoCas9 to provide the targeted endonuclease activity to cleave the cell DNA at a desired locus or gene of interest. A repair-oligo is also provided to the cell whereby following DNA cleavage, homologous recombination takes place in the cell with the repair-oligo so that either a deletion or substitution of nucleotides in the locus or gene of interest is achieved. Expression vectors and methods of using the vectors to achieve ThermoCas9 mediated gene editing are described whereby higher temperatures, e.g. greater than 30° C.