Abstract: The present invention encompasses engineered nucleases which recognize and cleave a recognition sequence within the int22h-1 sequence of a Factor VIII gene. The present invention also encompasses methods of using such engineered nucleases to make genetically-modified cells, and the use of such cells in a pharmaceutical composition and in methods for treating hemophilia A. Further, the invention encompasses pharmaceutical compositions comprising engineered nuclease proteins, nucleic acids encoding engineered nucleases, or genetically-modified cells of the invention, and the use of such compositions for treating of hemophilia A.

Abstract: Provided herein is the identification and use of the Cannabis Ubiquitin promoter and its use in promoting the expression of one or more heterologous nucleic acid fragments in a constitutive manner in Cannabis plants. The invention further discloses compositions, polynucleotide constructs, transformed host cells, plants and seeds containing the recombinant construct with the promoter, and methods for preparing and using the same.

Abstract: The present invention relates in part to nucleic acids, including nucleic acids encoding proteins, therapeutics and cosmetics comprising nucleic acids, methods for delivering nucleic acids to cells, tissues, organs, and patients, methods for inducing cells to express proteins using nucleic acids, methods, kits and devices for transfecting, gene editing, and reprogramming cells, and cells, organisms, therapeutics, and cosmetics 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, including cells present in vivo. Therapeutics comprising nucleic acids encoding gene-editing proteins are also described.

Abstract: A data collection hub and method wherein a controller is configured to receive data from a plurality of sensors sensing conditions related to the performance of operations on cells by at least one instrument. A database stores the data from the plurality of sensors and the controller is configured to compare data from the plurality of sensors for a past operation with the data for a more recent operation.

Abstract: The invention comprises a rapid in vitro method of assessing activity of an endonuclease, and a substrate therefor. Compositions, diagnostic methods, and kits are also disclosed.

Abstract: A hydrogel particle, comprising a matrix comprising a polymerized monomer, said matrix comprising a plurality of micropores and a plurality of macropores, and one or more immunostimulatory biomolecules selected from the group consisting of an anti-CD3 antibody or antigen-binding fragment thereof, an anti-CD28 antibody or antigen-binding fragment thereof, and combinations thereof.

Abstract: The invention relates generally to multispecific polypeptides that bind at least CD3, a second antigen, and a receptor of a T cell, such as a costimulatory receptor or an inhibitory receptor, in which the multispecific polypeptide constructs are able to engage CD3. In some embodiments, the multispecific polypeptide constructs bind a costimulatory receptor and provide costimulatory binding activity. In some embodiments, the multispecific polypeptide constructs bind an inhibitory receptor and block inhibitory activity. In some aspects, the multispecific polypeptides have constrained CD3 binding and bind to or engage CD3 only upon binding to the second antigen, such as a tumor associated antigen. In some embodiments, the multispecific polypeptides contain cleavable linkers that, when cleaved, result in dual effector functions. Also provided are methods of making and using these multispecific polypeptides in a variety of therapeutic, diagnostic and prophylactic indications.

Abstract: The present disclosure provides compounds and pharmaceutically acceptable salt thereof, and methods of using the same. The compounds and methods have a range of utilities as therapeutics, diagnostics, and research tools. In particular, the subject compositions and methods are useful for reducing signaling output of oncogenic proteins.

Abstract: The disclosure provides methods and reagents for preparing DNA libraries from biological materials for targeted sequencing. The approach can enhance the efficiency and sensitivity of targeted sequencing applications, such as liquid biopsy analyses to assess genetically driven conditions.

Abstract: Compounds of Formula I that inhibit AXL, and compositions containing the compound(s) and methods for synthesizing the compounds, are described herein. Also described are the use of such compounds and compositions for the treatment of a diverse array of diseases, disorders, and conditions, including cancer, that are mediated, at least in part, by AXL.

Abstract: The present disclosure encompasses methods of cancer immunotherapy, and particularly methods of allogeneic cellular immunotherapy, using particular lymphodepletion regimens in combination with particular populations of chimeric antigen receptor T cells.

Abstract: The present disclosure provides feeder hydrogel particles that can function to support the growth, proliferation, and/or activation of a target cell in culture. The present disclosure also provides methods of culturing target cells with feeder hydrogel particles.

Abstract: Provided herein are compositions and methods for the suppression of multi-drug resistant organisms. Provided herein are compositions and methods for treating diseases or disorders associated with bacterial colonization or treating diseases or disorders associated with an immune response induced by bacteria. Also provided herein are compositions and methods for suppressing colonization of the intestine of subject with oral microbiome bacteria.

Abstract: Provided herein are VHH-containing polypeptides that bind CLEC12a. Uses of the VHH-containing polypeptides are also provided.

Abstract: An apparatus for loading and imaging a microfluidic chip can comprise a housing having walls that define a vacuum chamber and a first receptacle disposed within the vacuum chamber, the first receptacle defining a space for receiving one or more microfluidic chips. The apparatus can also include a negative pressure source, a light source, and an optical sensor coupled to the housing. The negative pressure source can be configured to reduce pressure within the vacuum chamber, the light source can be positioned to illuminate at least a portion of the space for receiving the chip(s), and the optical sensor can be positioned to capture an image of at least a portion of the space for receiving the chip(s).

Abstract: Disclosed herein are highly sensitive immunoassays that utilize a capture/release mechanism to reduce non-specific binding and achieve detection with attomolar-level sensitivity. Kits that can be used for carrying out these highly sensitive immunoassays are also disclosed herein.

Abstract: This invention relates generally to molecules that specifically engage 41BB, a member of the TNF receptor superfamily (TNFRSF). More specifically, this invention relates to multivalent and multispecific molecules that bind at least 41BB.

Abstract: Provided herein are hydrogel beads that mimic live, dead, and apoptotic cells. The present disclosure also provides kits and compositions of hydrogel beads. The present disclosure further comprises methods of using the kits, compositions, and hydrogel beads to determine if a target cell sample includes one or more live, dead, or apoptotic cells.

Abstract: The present invention provides a DNA-targeting RNA comprising a single-guide RNA (sgRNA) and a ribonucleotide sequence rich in adenine ribonucleotide, and its use in gene editing, and a method for improving the efficiency of sgRNA-mediated gene editing, comprising a step of adding a ribonucleotide sequence rich in adenine ribonucleotide at 3? end of the sgRNA.

Abstract: A pulse generation system is disclosed. The pulse generation system includes a controller, an output terminal, and a plurality of pulse generator circuits. The controller is configured to cause a driving signal pulse to be transmitted to any selected one or more of the pulse generator circuits, and to cause the driving signal pulse to not be transmitted to any selected one or more other pulse generator circuits. Each of the pulse generator circuits is configured to generate an output voltage pulse at the output terminal in response to the driving signal pulse being transmitted thereto.