Abstract: The present disclosure provides improved genome editing compositions and methods for editing a human Wiskott-Aldrich syndrome gene. The disclosure further provides genome edited cells for the prevention, treatment, or amelioration of at least one symptom of WAS, including but not limited to, an immune system disorder, thrombocytopenia, eczema, X-linked thrombocytopenia (XLT), or X-linked neutropenia (XLN).

Abstract: The present invention relates to the discovery that etanercept reduces the rate of resistance to intravenous gamma globulin (IVIG) in subjects with acute Kawasaki disease (KD). In certain embodiments, the co-administration of etanercept and IVIG more effectively treats acute KD in subjects older than 12 months than IVIG alone. In other embodiments, the co-administration of etanercept and IVIG ameliorates coronary artery dilation in high risk subjects.

Abstract: Disclosed herein are methods of engineering a bi-specific T-cell expressing chimeric antigen receptors for promoting the in vivo expansion and activation of an effector cell and a second chimeric antigen receptor or TcR specific for a ligand on a tumor. Methods of administering to subjects in need, bi-specific chimeric antigen receptor bearing cells are also provided.

Abstract: A kit for resuscitating a subject, particularly a neonatal patient, is described. In an embodiment, the kit includes a syringe having a tip defining a male Luer taper fitting, and an adaptor defining a first end portion shaped to couple with the male Luer taper fitting and a second end portion shaped to cooperatively couple with an opening of an endotracheal tube.

Abstract: Provided herein are several monoclonal antibodies that activate the adhesion activity of human and mouse E-cadherin, including the amino acid sequences for the CDRs that define the binding domains of each monoclonal antibody. Also described are methods of making these antibodies, as well as biologically functional fragments and derivatives thereof; and methods of using them in the treatment, prevention, and/or amelioration of disease and conditions characterized by disruption of normal cell adhesion and/or cell junctions. Specifically contemplated are methods and compositions for the treatment of cancer metastasis as well as inflammatory conditions (such as inflammatory bowel disease and airway inflammation).

Abstract: The disclosure provides a panel of biomarkers that individually or in combination can indicate the presence of sepsis as distinguishable from other non-infection related inflammatory conditions. The disclosed biomarkers and related reagents and kits provide strategies for detecting, treating, and monitoring sepsis in subjects. In aspect, the disclosure provides a method for detecting sepsis, comprising contacting a biological sample obtained from the subject with an affinity reagent that specifically binds to one or more of the disclosed novel biomarkers, and detecting differential expression of the one or more biomarkers by detecting binding of the affinity reagent to the biomarker. The method can incorporate use of additional known biomarkers. The method can further comprise treating a subject determined to have sepsis. In some embodiments, the subject is a human subject less than 20 years old.

Abstract: An antimicrobial preventive netting is disclosed that includes one or more photosensitizers that are capable of generating a cloud of singlet oxygen on each surface and openings through the netting in response to incident light. A depth of the singlet oxygen cloud may be less than 0.7 centimeters from a netting surface. The netting may be supported by screens, enclosures or headgear. The netting can be made by dipping or soaking the netting in a solution of one or more photosensitizers.

Abstract: The present invention provides genetic tags operably linked to transgenes. The expression of the genetic tag allows identification, detection, selection, and ablation of cells expressing the transgene and the genetic tag. In some alternatives the genetically modified host cell comprises a transgene comprising a polynucleotide coding for a chimeric antigen receptor comprising a ligand binding domain, a polynucleotide comprising a spacer region, a polynucleotide comprising a transmembrane domain, and a polynucleotide comprising an intracellular signaling domain and a polynucleotide coding for a genetic tag.

Abstract: Provided herein is a system for inducible expression of a chimeric antigen receptor in cells, such as mammalian cells.

Abstract: Disclosed herein are polynucleotides having a plurality of thymine nucleotides and an endonuclease recognition site inserted therein, methods of engineering the polynucleotides having a plurality of thymine nucleotides and an endonuclease recognition site inserted therein, and methods of enhancing transcription, translation, and increasing stability of a polynucleotide.

Abstract: Systems, kits, and related methods of making fecal transplants are described. In an embodiment, the system comprises a sample collector defining a sample collection chamber shaped to collect a fecal sample from fecal matter; a sample processing module defining a sample port shaped to receive the sample collector and comprising: a channel positioned to receive at a proximal end of the channel the fecal sample from the sample collection chamber when the sample collector is received by the sample port; and a capsule fluidically coupled to a distal end of the channel adapted to receive a portion of the fecal sample passed therethrough; and a sample processing unit comprising: a housing defining an opening shaped to receive the sample processing module; and an actuator disposed in the housing and configured to urge the sample from the sample collection chamber through the channel to the capsule.

Abstract: An IV system can include a first flow regulator configured to restrict flow through a tube and a second flow regulator configured to restrict flow through the tube downstream of the first flow regulator. The first flow regulator can compress the tube a fixed amount to set an initial flow rate through the tube, while the second flow regulator includes a user-adjustable mechanical compression device that compresses the tube to a variable degree to fine-tune the flow rate through to a more precise flow rate that is lower than the initial flow rate. The flow regulators can utilize a purely mechanical means such that electricity is not required. Such systems can provide a simple, low-cost, and low-power way to provide precise flow control to patients, especially neonatal patient, in any environment. Systems can also include flow rate monitoring, alerting, and shutoff components.

Abstract: Methods of reducing or inhibiting Clostridium infection, treating intestinal dysbiosis associated with Clostridium infection, and preventing Clostridium colonization or Clostridium recolonization in a subject are disclosed. The methods include administering to the subject a whole foods exclusionary diet, such as a diet that excludes grains, sugars other than honey, and milk products other than hard cheeses and yogurt fermented for greater than 24 hours.

Abstract: Neurological implants whose surfaces have been chemically and covalently modified to impart beneficial properties to the neurological implants are described. The neurological implants possess improved biocompatibility compared to a corresponding neurological implant that lacks the chemical modification. Following implantation in a subject, the surface-modified neurological implants induce a lower-foreign body response, compared to a corresponding unmodified product.

Abstract: Some embodiments of the methods and compositions provided herein relate to transgenes comprising regulatory elements capable of inducing specific transcription of an operably-linked therapeutic payload in a cell in an in vivo microenvironment. In some embodiments, the regulatory elements are responsive to endogenous stimuli of the microenvironment. In some embodiments, the regulatory elements are response to stimuli from chimeric receptors in the cell.

Abstract: The present disclosure relates to methods of treating a patient with a cancer by administering to the patient a composition comprising CAR T cells wherein the CAR T cells comprise a CAR and the CAR comprises an E2 anti-fluorescein antibody fragment, and administering to the patient a small molecule linked to a targeting moiety by a linker. The disclosure also relates to compositions for use in such methods.

Abstract: The present disclosure relates to biofragment compositions that comprise bioparticle fragments and at least one heterologous antigen-binding molecule. In some embodiments, the biofragment is typically derived from a larger, intact bioparticle that express the at least one heterologous antigen-binding molecule at the surface, and the biofragment has increased solubility to facilitate assays for antigen detection. The disclosure also relates the related methods of using and making the biofragment compositions, as well as systems and devices implementing the biofragment compositions. In some embodiments, the related methods, systems and devices do not require additional detection reagents, such as animal derived detection antibodies.

Abstract: The present invention relates to the discovery that etanercept reduces the rate of resistance to intravenous gamma globulin (IVIG) in subjects with acute Kawasaki disease (KD). In certain embodiments, the co-administration of etanercept and IVIG more effectively treats acute KD in subjects older than 12 months than IVIG alone. In other embodiments, the co-administration of etanercept and IVIG ameliorates coronary artery dilation in high risk subjects.

Abstract: A CD19-OR-CD20 chimeric antigen receptor (CAR) protein construct is provided. Also provided are nucleic acids encoding the CD19-OR-CD20 CAR; and methods of use, e.g. in the treatment of B cell malignancies. The CD19-OR-CD20 CAR of the invention is a bispecific CAR that can trigger T-cell activation upon detection of either CD19 or CD20 (or both). It is a single molecule that confers two-input recognition capability upon human T cells engineered to stably express this CAR.

Abstract: This invention relates to a prognostic method for determining the risk of an asymptomatic human subject with latent tuberculosis (TB) infection or apparent latent TB infection and/or after suspected exposure to TB progressing to active tuberculosis disease comprising the steps of quantifying and computationally analysing relative abundances of a collection of pairs of gene products (“TB biomarkers”) derived from a sample obtained from the subject. The invention further relates to a collection of TB biomarkers that generates a transcriptomic signature of risk for prediction of the likelihood of an asymptomatic human subject with latent TB infection and/or after suspected exposure to TB progressing to active tuberculosis disease. Furthermore, a kit comprising gene-specific primers or oligonucleotide probes for the detection of pairs of TB biomarkers that generates a prognostic signature of risk for use with the method of the invention is described.