Abstract: Provided are methods for preventing or ameliorating toxicity caused by or due to a therapy, such as an immunotherapy or a cell therapy, by pre-emptive or early administration toxicity-targeting agent(s). In some embodiments, the therapy is a cell therapy in which the cells generally express recombinant receptors such as chimeric receptors, e.g., chimeric antigen receptors (CARs) or other transgenic receptors such as T cell receptors (TCRs). Features of the methods, including the timing of the administration of the agents or treatments for toxicity, provide various advantages, such as lower toxicity while maintaining persistence and efficacy of the administered cells.

Abstract: The present application relates to compositions comprising fusion proteins and cells expressing the proteins. The application further relates to methods of using the fusion proteins, cells, and compositions for modulating cell signaling and for selective expansion of cells.

Abstract: A system and method for predicting risk of violence for an individual (primarily school violence, but not limited to school violence) performs the following steps: (a) receiving responses to questions from an individual; (b) extracting by a computerized annotator words or phrases from the questions and responses; (c) assigning by the annotator extracted word(s) or phrase(s) to at least one of a plurality of pre-defined categories; and (d) automatically identifying and scoring words or phrases that could be classified into the pre-defined categories by a trained machine-learning engine to produce a score reflecting relative risk of violence by the individual. The pre-defined categories include: expression of violent acts or thoughts of the individual; expression of negative feelings, thoughts or acts of others; expression of negative feelings, thoughts or acts of the individual; expression of family discord or tragedies; and expression of protective factors.

Abstract: Methods and compositions disclosed herein generally relate to methods of improving clinical and economic outcomes to address adverse effects related to anesthesia, analgesics, opioids, and inadequate pain relief. Embodiments of the invention relate to the association between genes, specific polymorphisms of genes, and non-genetic factors with inadequate pain relief and anesthesia-, analgesic, and/or opioid-related adverse effects. Embodiments of the invention can be used to determine and manage patient risk factors for development of adverse perioperative effects and can allow for personalized anesthesia and pain management for improvement of pain control and reduction of anesthesia-, analgesic-, and opioid-related adverse outcomes. These methods and compositions apply to non-surgical pain management with opioids.

Abstract: Methods of breaking down a biofilm or inhibiting, preventing or treating a microbial infection that produces a biofilm are disclosed, which involves administration of a polypeptide that has one or more modified HMG-box 1 domains to a subject suffering from the infection or having the biofilm. By competing with microbial proteins that bind to DNA scaffold in the biofilm, these polypeptides destabilize the biofilm leading to destruction and removal of the biofilm by the immune system.

Abstract: The present invention relates to Adeno-associated virus type 9 methods and materials useful for intrathecal delivery of polynucleotides. Use of the methods and materials is indicated, for example, for treatment of lower motor neuron diseases such as SMA and ALS as well as Pompe disease and lysosomal storage disorders. It is disclosed that administration of a non-ionic, low-osmolar contrast agent, together with a rAAV9 vector for the expression of Survival Motor Neuron protein, improves the survival of SMN mutant mice as compared to the administration of the expression vector alone.

Abstract: Embodiments disclosed herein provide compositions, methods, and uses for recombinant vectors encoding Zika virus (ZIKV) protein subunits, and immunogenic compositions thereof. Certain embodiments provide recombinant vectors encoding ZIKV nonstructural protein 1 (NS 1), and optionally, ZIKV envelope (E) protein and premembrane (prM) protein. Other embodiments provide expression cassettes comprising a promoter operably linked to a polynucleotide that encodes the ZIKV NS 1 protein, and optionally ZIKV E and prM proteins. In some embodiments, the disclosed expression cassettes can be incorporated into a vector to produce a recombinant vector. Also provided are immunogenic compositions comprising one or more recombinant vectors described herein, and methods for inducing an immune response against ZIKV in a subject comprising administering to the subject an immunologically effective dose of an immunogenic composition of the present disclosure.

Abstract: The disclosure relates to methods for pain management in the perioperative context, particularly through the use of the DNA methylation status of the human OPRM1 gene as a biomarker for increased susceptibility to perioperative pain, and related methods and compositions.

Abstract: The present invention relates to Adeno-associated virus type 9 methods and materials useful for intrathecal delivery of polynucleotides. Use of the methods and materials is indicated, for example, for treatment of lower motor neuron diseases such as SMA and ALS as well as Pompe disease and lysosomal storage disorders. It is disclosed that administration of a non-ionic, low-os-molar contrast agent, together with a rAA9 vector for the expression of Survival Motor Neuron protein, improves the survival of SMN mutant mice as compared to the administration of the expression vector alone.

Abstract: The invention relates to an insertable patient table for a Magnetic Resonance Imaging scanner, wherein the patient table has a length of up to ± 20 % of the length of the bore of the main magnet of the scanner and is adapted for pediatric and/or small patients, and wherein the patient table is adapted to be inserted into the bore of the main magnet without having a stand standing on the floor of an examination room. The invention further relates to a Magnetic Resonance Imaging scanner comprising an insertable patient table and a method for positioning a small and/or pediatric patient within the bore of a main magnet of a Magnetic Resonance Imaging scanner.

Abstract: The present invention provides an advancement in the art of cardiac pacemakers. The invention provides a novel and unobvious pacemaker system that comprises at least one pacemaker and that is, to a large extent, self-controlled, allows for long-term implantation in a patient, and minimizes current inconveniences and problems associated with battery life. The invention further includes a mechanism in which at least two pacemakers are implanted in a patient, and in which the pacemakers communicate with each other at the time of a given pacing or respiratory event, without any required external input, and adjust pacing parameters to respond to the patient's need for blood flow. The invention further provides a novel design for a pacemaker in which the pacemaker electrode is connected to the pacemaker body by a lead that is configured to allow the pacemaker to lie parallel to the epicardial surface and to reduce stress on the pacemaker and heart tissue.

Abstract: The invention provides for recombinant AAV vectors comprising a miniaturized human micro-dystrophin gene and methods of using the recombinant vectors to reduce or prevent fibrosis in subjects suffering from muscular dystrophy.

Abstract: Zebrafish are a powerful model for investigating cardiac repair due to their unique regenerative abilities, scalability, and compatibility with many genetic tools. However, characterizing the regeneration process in live adult zebrafish hearts has proved challenging because adult fish are opaque and explanted hearts in conventional culture conditions experience rapid declines in morphology and physiology. To overcome these limitations, we fabricated a fluidic device for culturing explanted adult zebrafish hearts with constant media perfusion that is also compatible with live imaging. Unlike hearts cultured in dishes for one week, the morphology and calcium activity of hearts cultured in the device for one week were largely similar to freshly explanted hearts.

Abstract: This disclosure provides new genetic targets, diagnostic methods, and therapeutic treatment regimens for multiple autoimmune disorders, including pediatric autoimmune disorders that are co-inherited and genetically shared. The disclosure, for example, provides methods of diagnosing or determining a susceptibility for one or more autoimmune diseases and methods of determining treatment protocols for patients with one or more autoimmune diseases based on determining if the patients have genetic alterations in particular genes.

Abstract: Disclosed are methods of making a genetically cell that expressed FOXP3 and methods of treatment. In some embodiments, the method can providing a first nucleotide sequence, wherein the first nucleotide sequence comprises a coding strand, the coding strand comprising one or more regulatory elements and a FOXP3 gene or portion thereof providing a nuclease and performing a gene editing process on the first nucleotide sequence, which edits said one or more regulatory elements, and optionally edits the FOXP3 gene or portion thereof. Methods of treating a subject suffering from an autoimmune disease and subjects suffering the effects of organ transplantation are also provided.

Abstract: Decellularized matrix microspheres comprising a polymeric material and a donor tissue are provided. Also disclosed are structures containing a plurality of decellularized matrix microspheres incorporating a first polymer and a donor tissue; and a second polymer, wherein the decellularized matrix microspheres and the second polymer are in the form of a filament. Methods of treating a tissue injury employing the matrix microspheres and structures described as well as their methods of manufacture are also provided.

Abstract: Aspects of the invention described herein concern the incorporation of a FOXP3 cDNA (e.g., full-length human codon-optimized cDNA) into a FOXP3 gene or a non-FOXP3 locus so as to provide constitutive or regulated FOXP3 expression in a primary human CD34+ cells or cells derived from edited CD34+ cells. In some embodiments, guide RNA sequences that are directed to FOXP3, AAVS1, or other candidate loci are used for CRISPR/Cas9-mediated gene regulation, and gene delivery cassettes for HDR based gene-modification are provided.

Abstract: Embodiments provide a deep learning framework to accurately segment liver and spleen using a convolutional neural network with both short and long residual connections to extract their radiomic and deep features from multiparametric MRI. Embodiments will provide an “ensemble” deep learning model to quantify biopsy derived liver fibrosis stage and percentage using the integration of multiparametric MRI radiomic and deep features, MRE data, as well as routinely available clinical data. Embodiments will provide a deep learning model to quantify MRE-derived liver stiffness using multiparametric MRI, radiomic and deep features and routinely-available clinical data.

Abstract: Extracorporeal membrane oxygenation systems and methods of use are disclosed. Several publications and patent documents are cited throughout the specification in order to describe the state of the art to which this invention pertains. Full citations of these references can be found throughout the specification. Each of these citations is incorporated herein by reference as though set forth in full.

Abstract: Provided herein are engineered red blood cells expressing rare blood antigen group profiles, and methods of making use the same, are described. Also provided are recombinant reagent red blood cells that express or lack the expression of at least one protein (e.g., a blood group antigen) on its surface and uses thereof.