Abstract: Provided herein are programmable nucleases and methods of genome editing and detection of nucleic acids with said programmable nuclease. In some embodiments, the programmable nuclease is a programmable nickase.

Abstract: Disclosed herein are compositions and pharmaceutical formulations that comprise a binding moiety conjugated to a modified polynucleic acid molecule and a polymer. Also described herein include methods for treating a disease which utilize a composition or a pharmaceutical formulation comprising a binding moiety conjugated to a polynucleic acid molecule and a polymer.

Abstract: Provided herein are engineered variants of archaeal polymerases that exhibit exonuclease-minus activity, enhanced thermostability, enhanced incorporation of 3? modified nucleotides, improved uracil-tolerance and/or reduce sequence-specific errors in polymerase-catalyzed nucleotide binding and extension reactions relative to wild type polymerase enzymes. Also provided are uses of the engineered polymerases for forming complexed polymerases and forming binding complexes, and uses for conducting nucleic acid sequencing reactions.

Abstract: The present disclosure encompasses engineered meganucleases that bind and cleave recognition sequences within a dystrophin gene. The present disclosure also encompasses methods of using such engineered meganucleases to make genetically modified cells. Further, the disclosure encompasses pharmaceutical compositions comprising engineered meganuclease proteins, or polynucleotides encoding engineered meganucleases of the disclosure, and the use of such compositions for the modification of a dystrophin gene in a subject, or for treatment of Duchenne Muscular Dystrophy.

Abstract: The present invention relates in part to nucleic acids encoding proteins, therapeutics comprising nucleic acids encoding proteins, methods for inducing cells to express proteins using nucleic acids, methods, kits and devices for transfecting, gene editing, and reprogramming cells, and cells, organisms, and therapeutics produced using these methods, kits, and devices. Methods and products for altering the DNA sequence of a cell are described, as are methods and products for inducing cells to express proteins using synthetic RNA molecules. Therapeutics comprising nucleic acids encoding gene-editing proteins are also described.

Abstract: The present invention provides novel pharmaceutical compositions comprising -(4-Chloro-2-(morpholin-4-yl)thiazol-5-yl)-7-(1-ethylpropyl)-2,5-dimethylpyrazolo(1,5-a)pyrimidine and methods of using the same for the treatment of Congenital adrenal hyperplasia (CAH).

Abstract: Provided herein are acute myeloid leukemia antigen targets for chimeric receptors and methods of using same.

Abstract: The present disclosure relates to human milk compositions and methods of making and using the same. In particular, the disclosure features methods of using human milk compositions to feed subjects before and/or after surgery or medical operations and that are useful in promoting recovery.

Abstract: The application relates to a dual cytokine fusion protein composition, pharmaceutical composition, and/or formulation thereof comprising IL-10 or IL-10 variant molecules fused to a single chain variable fragment scaffolding system and a second cytokine, where the second cytokine is linked in the hinge region of the scFv. The application also relates to methods of using the dual cytokine fusion protein composition for treating cancer, inflammatory diseases or disorders, and immune and immune mediated diseases or disorders.

Abstract: The present embodiments relate to an apheretic system incorporating one or more centrifuge devices. A biological fluid containing cells (or another fluid) can be obtained and directed to a centrifuge device to separate particles by a particle size and/or a flow rate. The centrifuge device can include a center-axis centrifuge, an off-center centrifuge, and/or a hydro-cyclone centrifuge device. The separated particles can be directed/drawn into either an outlet to a return or into a collection container for further testing.

Abstract: The present embodiments relate to an apheretic system incorporating one or more centrifuge devices. A biological fluid containing cells (or another fluid) can be obtained and directed to a centrifuge device to separate particles by a particle size and/or a flow rate. The centrifuge device can include a center-axis centrifuge, an off-center centrifuge, and/or a hydro-cyclone centrifuge device. The separated particles can be directed/drawn into either an outlet to a return or into a collection container for further testing.

Abstract: The present invention is directed to a humanized BCMA single-chain variable fragment (scFv), comprising VH having the amino acid sequence of SEQ ID NO: 3 and VL having the amino acid sequence of SEQ ID NO: 5. The present invention is also directed to a BCMA chimeric antigen receptor fusion protein comprising from N-terminus to C-terminus: (i) a single-chain variable fragment (scFv) of the present invention, (ii) a transmembrane domain, (iii) at least one co-stimulatory domains, and (iv) an activating domain. This humanized BCMA-CAR-T cells have specific killing activity against BCMA-positive tumor cells.

Abstract: Method and apparatus for performing a plaque detection by using above focus and below focus images. Plaque is also detected by a method and apparatus using test and training data captured on an imaging system, building a new model for a specific virus/cell/protocol type to detect plaques, using the models in runtime systems to detect plaques and augmenting the models based on automatically calculated false positive and false negative counts and percentages taken from test runs and/or runtime data.

Abstract: Disclosed herein are polynucleic acid molecules, pharmaceutical compositions, and methods for treating Facioscapulohumeral muscular dystrophy.

Abstract: Described herein are humanized anti-TL1A antibodies and pharmaceutical compositions for the treatment of inflammatory bowel disease (IBD), such as Crohn's Disease (CD) and ulcerative colitis (UC).

Abstract: Methods and systems are provided for synchronizing image capture at a multi-detector imaging system. In one example, a method includes coordinating cycling of each microscope assembly of the multi-detector imaging system through a selection of illumination channels, each microscope assembly configured to obtain an image of a portion of one of more than one microplate wells simultaneously, to generate complete images of the more than one microplate wells concurrently.

Abstract: The invention provides a method for genetic analysis in individuals that reveals both the genetic sequences and chromosomal copy number of targeted and specific genomic loci in a single assay. The present invention further provide methods for the sensitive and specific detection of target gene sequences and gene expression profiles.

Abstract: The disclosure relates to methods for colonizing a microbiome, by administering pharmaceutical compositions comprising a purified bacterial mixture to the subject. Also provided are methods for treating and/or preventing inflammatory bowel disease, methods for reducing the risk of inflammatory bowel disease and/or reducing the occurrence of inflammatory bowel disease in a subject by administering pharmaceutical compositions comprising a purified bacterial mixture to the subject. Also provided are methods for treating and/or preventing graft versus host disease in a subject by administering pharmaceutical compositions comprising a purified bacterial mixture to the subject.

Abstract: A clustered regularly interspaced short palindromic repeat (CRISPR)-associated complex for adaptive antiviral defence (Cascade); the Cascade protein complex comprising at least CRISPR-associated protein subunits Cas7, Cas5 and Cas6 which includes at least one subunit with an additional amino acid sequence possessing nucleic acid or chromatin modifying, visualising, transcription activating or transcription repressing activity. The Cascade complex with additional activity is combined with an RNA molecule to produce a ribonucleoprotein complex. The RNA molecule is selected to have substantial complementarity to a target sequence. Targeted ribonucleoproteins can be used as genetic engineering tools for precise cutting of nucleic acids in homologous recombination, non-homologous end joining, gene modification, gene integration, mutation repair or for their visualisation, transcriptional activation or repression.

Abstract: An apparatus attaches to a microscope slide to form an enclosed fluidic chamber with input and output ports, where a tissue or cell sample on the slide can be processed using techniques such as immunohistochemical staining. Flow of reagents within the chamber can be laminar and highly uniform across the sample surface to achieve staining that is free of gradients. The sample can be imaged prior to, during, or after any given processing step. Methods for staining and imaging a sample can be implemented through one or more rounds of labeling, label removal or erasure and imaging. Samples can be imaged many times to achieve very high multiplexing levels. Samples can be placed on an imaging apparatus at various times and removed from the imaging apparatus for certain steps.