Abstract: The present invention provides methods and compositions for specific activation of inflammatory responses in dendritic cells (DCs). 1-palmitoyl-2-arachidonyl-sn-glycero-3-phosphorylcholine (PAPC) and its oxidized variant (oxPAPC) were identified to promote DC-mediated immunity, and are provided as adjuvants in immunostimulatory compositions, including vaccines.

Abstract: Compositions and methods for administration of local anesthetics that are delivered by a single injection and enable repeated on-demand or high influx analgesia over extended periods have been developed. Pharmaceutical compositions including an effective amount of one or more sodium channel blockers including site 1 sodium channel blockers, optionally one or more alpha-2-adrenergic agonists, which are optionally encapsulated in liposomes, particles or microbubbles, and one or more triggerable elements are provided. The triggerable elements allow delivery of the encapsulated anesthetic drugs when an appropriate triggering stimuli are applied. Exemplary triggering agents or stimuli include near-infrared irradiation, UV- and visible light, ultrasound and magnetic field. In one embodiment, ultrasound is used to trigger a burst of microbubbles to enhance penetration of local anesthetic.

Abstract: Covalently modified alginate polymers, possessing enhanced biocompatibility and tailored physiochemical properties, as well as methods of making and use thereof, are disclosed herein. The covalently modified alginates are useful as a matrix for the encapsulation and transplantation of cells. Also disclosed are high throughput methods for the characterizing the biocompatibility and physiochemical properties of modified alginate polymers.

Abstract: Described herein are methods and compositions related to the modulation of progranulin expression or activity in the brain for the treatment of neurodegenerative diseases.

Abstract: The present invention relates generally to methods, compositions and kits for treatment of ribosomal disorders and ribosomopathy, e.g. Diamond Blackfan anemia (DBA). In some embodiments, the invention relates to methods for the use of calmodulin inhibitors and calcium channel blockers for treatment of ribosomal disorders and ribosomopathy, e.g. Diamond Blackfan anemia (DBA).

Abstract: Provided herein are cyclic prosaposin peptides and compositions and uses thereof. Exemplary uses include use in the treatment of cancer or in the treatment of inflammatory diseases or disorders.

Abstract: A pipetting device includes a housing, a valve assembly, and an actuator assembly. The housing includes a tip for receiving a capillary tube. The valve assembly includes a shuttle valve having a filling position and a dispensing position and a valve rod. The actuator assembly includes an actuator, a valve trigger, a piston mount, and an indexing mechanism. The actuator extends from the housing and has a push button coupled to a push rod. The valve trigger being configured to engage the valve assembly and includes an aperture configured to receive the piston mount therein. The indexing mechanism is configured to index a rotated position of the push button to a predefined volume of dispensed fluid.

Abstract: Methods are described for the detection of low frequency genetic variants, such as somatic mosaic variants. The methods comprise parallel amplification reactions of a target nucleic acid sequence to generate overlapping amplicons, pooled sequencing of the amplicons, and demultiplexed detection of low frequency variants.

Abstract: A plug is configured to maintain position within a Eustachian tube of a patient. The plug includes a proximal portion and a distal portion. The proximal portion includes a first body dimensioned bear radially outwardly against the Eustachian tube of the patient. The distal portion includes an anchoring assembly that is configured to lock the plug in the Eustachian tube of the patient. The plug may be formed of a bioabsorbable material.

Abstract: An instrument port includes a port body, a bulb disposed at the distal end of the port body, a base body disposed at the proximal end of the port body, a hollow cylindrical body mechanically coupled to the base body, and a plurality of connecting rods disposed parallel to the port body. Each connecting rod includes a distal rod portion that is mechanically coupled to a respective distal-facing exterior surface of a bulb flange and a proximal rod portion that is mechanically coupled to a respective proximal-facing surface of the base body. A spring applies a spring force against the port body to press the port body into the bulb to mechanically secure the bulb to the base body.

Abstract: Described herein are emulsion compositions comprising fish oil and medium-chain triglycerides (MCT) and/or omega-3 fatty acids and medium-chain triglycerides (MCT). In some embodiments, the compositions further comprise, e.g., alpha-tocopherol, or one or more fatty acids. Further provided herein are methods of using the compositions, e.g., to provide nutrion, e.g., parenteral nutrition to a subject and/or to treat or prevent certain conditions described herein.

Abstract: Among other things, a respiratory support score is determined. Operating parameters of one or more respiratory support devices associated with a patient are received. Based on the operating parameters, a respiratory support type associated with the patent is determined. A score range for the respiratory support type is received, and a respiratory support score within the range is determined based on at least a subset of the operating parameters. In another aspect, a medication efficacy score is determined. A time point corresponding to a clinical action performed on a patient is identified, the clinical action including administration of a drug at a particular dosage. Parameters indicative of a clinical condition of the patient are tracked for a time range that includes the time point corresponding to the clinical action. Based on the parameters, a metric indicative of an efficacy of the clinical action performed on the patient is determined.

Abstract: The invention provides methods of simultaneously detecting one or more biomarkers associated with one or more platelets in a platelet sample by contacting the sample with one or more metal-tagged probes or mixtures thereof; washing the sample to remove unbound probes; and analyzing the sample by mass cytometry to simultaneously detect binding of the one or more metal-tagged probes or mixtures thereof to one or more biomarkers associated with the one or more platelets. Compositions, panels and kits for use with the methods described herein are also provided.

Abstract: The present invention features compositions and methods featuring CD82 for treating muscular dystrophies and related disorders. In one aspect, the invention provides a method of preserving or increasing muscle function in a dystrophic cell, the method involving contacting the cell with a CD82 polypeptide or a polynucleotide encoding a CD82 polypeptide.

Abstract: Various aspects and embodiments of the present invention are directed to methods of treating a subject having an ocular condition, methods of isolating ocular stem cells, methods of selecting and/or producing ocular grafts for transplantation, and methods of promoting ocular cell regeneration as well as to grafts and preparations containing isolated ocular stem cells characterized by the expression of ABCB5 on their cell surface.

Abstract: Provided herein are Toll-like receptor 2 (TLR2) agonists for use in enhancing human immune response and/or as adjuvants in vaccines. The TLR2 agonists include thiophenes, imidazoles, or phenyl-containing compounds, which may be compounds of Formulae (I), (II), (III), and pharmaceutically acceptable salts, solvates, hydrates, polymorphs, co-crystals, tautomers, stereoisomers, isotopically labeled derivatives, prodrugs, and compositions thereof. The compounds described herein are used as enhancers of an immune response (e.g., innate and/or adaptive immune response), and are useful in treating and/or preventing a disease, as adjuvants in a vaccine for the disease, (e.g., proliferative disease, inflammatory disease, autoimmune disease, infectious disease, or chronic disease). Also provided in the present disclosure are pharmaceutical compositions, kits, methods, and uses including or using a compound described herein.

Abstract: The present embodiments provide for an S. aureus (SA) Multiple Antigen Presenting System (MAPS) immunogenic composition comprising an immunogenic polysaccharide which induces an immune response, where at least one S. aureus (SA) peptide or polypeptide antigen is associated to the immunogenic polysaccharide by complementary affinity molecules. In some embodiments, the immunogenic polysaccharide can be an antigenic capsular polysaccharide of a Type 5 or Type 8 from S. aureus, or alternatively, a different immunogenic capsular or noncapsular polysaccharide, and where the protein or peptide SA antigens are indirectly linked via an affinity binding pair. The present SA-MAPS immunogenic compositions can elicit both humoral and cellular immune responses to the immunogenic polysaccharide and one or multiple SA antigens at the same time.

Abstract: The invention provides compositions and methods for allele specific gene editing. In particular, the invention provides methods and compositions for treating dominant progressive hearing loss by selectively inactivating a dominant mutation in TMC1.

Abstract: Antibodies, and antigen-binding fragments thereof, that specifically bind to Cluster of Differentiation 1a (CD1a) are provided. Embodiments include uses, and associated methods of using the antibodies, and antigen-binding fragments thereof.

Abstract: The present invention is based, in part, on the identification of novel methods for defining predictive biomarkers of response to anti-cancer drugs.