Abstract: RNA editing tools for use in systems designed to measure RNA in vivo and manipulate specific cell types are disclosed herein. An RNA sensor system comprising a) a single-stranded RNA (ssRNA) sensor comprising a stop codon and a payload; optionally wherein the ssRNA sensor further comprises a normalizing gene; and b) an adenosine deaminase acting on RNA (ADAR) deaminase; wherein the sensor is capable of binding to a ssRNA target to form a double-stranded RNA (dsRNA) duplex that becomes a substrate for the ADAR deaminase; wherein the substrate comprises a mispairing within the stop codon; and wherein the mispairing is editable by the ADAR deaminase, which editing can effectively remove the stop codon so as to enable translation and expression of the payload. A method of quantifying ribonucleic acid (RNA) levels using the RNA sensor system is also disclosed.

Abstract: Methods for quantifying DNA methylation that may be utilized for screening for diseases (e.g., cancer), diagnosing diseases (e.g., cancer type), monitoring progression of a disease, and monitoring response to a therapeutic treatment.

Abstract: Described herein are compositions comprising subsets of monocyte having distinct functional properties and methods for using the same to treat infectious disease.

Abstract: Provided herein are pluripotent stem cells comprising particular combinations of transcription factor for the production of oligodendrocyte progenitor cells (OPCs). Also provided herein are methods of producing the pluripotent stem cells and methods of using the pluripotent stem cells to produce (OPCs) and oligodendrocytes.

Abstract: Disclosed herein are methods, systems, and devices for oral drug delivery. The method generally involves orally administering a drug delivery device to the subject in need of treatment and triggering iontophoresis. The drug delivery device includes one or more active agents for delivery to the subject. The method can include the step of delivering the active agent to the intestinal mucosa after iontophoresis is triggered at the site, or simultaneously as iontophoresis is applied at the site. After the active agent(s) are delivered, the drug delivery device can be released from the intestinal mucosa. The iontophoresis can be performed for a period of time and at an electrical current that is effective to improve permeability of the one or more active agents across the intestine compared to orally administering the same drug delivery device in the absence of iontophoresis.

Abstract: The present invention generally relates to droplets and/or emulsions, such as multiple emulsions. In some cases, the droplets and/or emulsions may be used in assays, and in certain embodiments, the droplet or emulsion may be hardened to form a gel. In some aspects, a heterogeneous assay can be performed using a gel. For example, a droplet may be hardened to form a gel, where the droplet contains a cell, DNA, or other suitable species. The gel may be exposed to a reactant, and the reactant may interact with the gel and/or with the cell, DNA, etc., in some fashion. For example, the reactant may diffuse through the gel, or the hardened particle may liquefy to form a liquid state, allowing the reactant to interact with the cell. As a specific example, DNA contained within a gel particle may be subjected to PCR (polymerase chain reaction) amplification, e.g., by using PCR primers able to bind to the gel as it forms. As the DNA is amplified using PCR, some of the DNA will be bound to the gel via the PCR primer.

Abstract: Described in embodiments herein are thiol-ene polymeric compositions that exhibit photo-induced, reversible switching between a solid (e.g., elastomeric) state and a liquid (e.g., flowable) state. The thiol-ene polymeric compositions may comprise a vinyl oligomer comprising at least two vinyl groups, a thiol oligomer comprising at least two thiol groups, and a Type I photoinitiator. In some embodiments, the composition comprises an excess of thiol groups relative to vinyl groups (e.g., a ratio of thiol groups to vinyl groups in the composition is at least 3:1). Reversible switching between the solid state and the liquid state may be induced through exposure of the composition to electromagnetic radiation (e.g., ultraviolet (UV) radiation). In some cases, reversible switching may be induced by a relatively low amount of energy (e.g., about 1 J/cm2 or less).

Abstract: The specification provides programmable base editors that are capable of introducing a nucleotide change and/or which could alter or modify the nucleotide sequence at a target site in mitochondrial DNA (mtDNA) with high specificity and efficiency. Moreover, the disclosure provides fusion proteins and compositions comprising a programmable DNA binding protein (e.g., a mitoTALE, a mitoZFP, or a CRISPR/Casp) and double-stranded DNA deaminase that is capable of being delivered to the mitochondria and carrying out precise installation of nucleotide changes in the mtDNA. The fusion proteins and compositions are not limited for use with mtDNA, but also may be used for base editing of any double-stranded target DNA.

Abstract: Aspects of the disclosure provide nucleic acids and compositions comprising gene regulatory elements (GREs) for specific expression in nociceptor cells. Other aspects of the disclosure relate to the use of vectors and compositions comprising the gene regulatory elements for treating or managing pain and other neurological diseases in a subject in need thereof.

Abstract: Provided are methods of treating diabetes and/or obesity in a subject in need thereof, and methods of increasing the amount of cholic acid-7-sulfate (CA7S) in a subject. Further provided herein are methods of administering CA7S to a subject. Also provided are compositions and kits comprising cholic acid-7-sulfate, or a salt thereof for use in the treatment of diabetes and/or obesity.

Abstract: Provided are compositions and methods for treating diseases associated with expression of mesothelin comprising administering a cell that expresses a chimeric antigen receptor (CAR) specific to mesothelin in combination with a PD-L1 inhibitor.

Abstract: A device for simulating a function of a tissue includes a first structure, a second structure, and a membrane. The first structure defines a first chamber. The first chamber includes a matrix disposed therein and an opened region. The second structure defines a second chamber. The membrane is located at an interface region between the first chamber and the second chamber. The membrane includes a first side facing toward the first chamber and a second side facing toward the second chamber. The membrane separates the first chamber from the second chamber.

Abstract: A multi-segment reinforced actuator includes (a) a soft actuator body that defines a chamber and (b) a plurality of distinct reinforcement structures on or in respective segments of the soft actuator body. First and second reinforcement structures are respectively configured to produce a first and second actuation motions, respectively, in first and second segments of the soft actuator body when fluid flows into or out of the chamber. The actuation motions are selected bending, extending, expansion, contraction, twisting, and combinations thereof; and the first actuation motion differs from the second actuation motion. The actuator can be used, e.g., to facilitate bending of the thumb with corresponding bending, extending, expansion, contraction, and twisting actuation motions.

Abstract: In some embodiments, a dielectric elastomer device may include at least first and second dielectric elastomer layers, and a first layer of conductive particles disposed between the first and second dielectric elastomer layers and forming a first electrode of the device, wherein portions of the second dielectric elastomer layer are directly bonded with portions of the first dielectric elastomer layer through the first layer of the conductive particles. The dielectric elastomer layer may, for example, comprise a cured acrylic elastomer precursor with an additive including urethane, polybutadiene, or silicone. Electrodes in different layers may be interconnected by infusing a liquid or semi-liquid conductive material in contact with each of a plurality of the electrodes of the actuator or sensor device, and solidifying the conductive material to form a conductive path that interconnects the plurality of electrodes.

Abstract: The invention provides integrated Organ-on-Chip microphysiological systems representations of living Organs and support structures for such microphysiological systems.

Abstract: Disclosed herein are cells and populations of cells comprising a genome in which the B2M gene has been edited to eliminate surface expression of MHC Class I protein in the cells or population of cells, and methods for allogeneic administration of such cells to reduce the likelihood that the cells will trigger a host immune response when the cells are administered to a subject in need of such cells.

Abstract: The present disclosure relates to a microfluidic devices and methods for culturing bone marrow cells. Aspects include methods of preparing microfluidic devices and culturing bone marrow cells with the microfluidic devices. In some aspects, a method includes providing a microfluidic device having an upper chamber, a lower chamber, and a porous membrane separating the upper chamber from the lower chamber. The method further includes seeding walls of the lower chamber and a bottom surface of the membrane with endothelial cells. The method further includes providing a matrix within the upper chamber. The matrix includes fibrin gel and bone marrow cells. The method further includes filling or perfusing the upper chamber with a media.

Abstract: The present disclosure provides, in some embodiments, methods and compositions for single-molecule detection.

Abstract: The present disclosure provides compositions and methods for treating neurodevelopmental disorders, such as Rett syndrome and cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder.

Abstract: A terahertz device for detecting or emitting or for both detecting and emitting electromagnetic waves in the terahertz frequency range. The terahertz device comprises: a first waveguide branch and a second waveguide branch, the first and second waveguide branches being configured to allow optical signals to propagate through them, the first and second waveguide branches being nonlinear dielectric elements with a thickness of at most 500 micrometres; and an antenna arrangement comprising a set of antennas for capturing and/or emitting electromagnetic waves in the terahertz frequency range, the antennas being placed along at least one of the waveguide branches in an immediate vicinity of the respective waveguide branch and/or around the respective waveguide branch to at least partially enclose the respective waveguide branch in a respective antenna gap of the respective antenna.