Abstract: Compositions and methods for treating FSHD and for identifying agents useful for the treatment of FSHD.

Abstract: The present disclosure provides compounds of Formula (I), 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 and/or modifiers 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), or as stand alone anti-infective or immune response modifying agents. Also provided in the present disclosure are pharmaceutical compositions, kits, methods, and uses including or using a compound described herein.

Abstract: The invention features methods and compositions that are useful for the treatment of diseases, disorders, conditions, or injuries characterized by insufficient myelination. The methods involve administering GPR17 antagonists and microglia inhibitors or ablation agents.

Abstract: The present disclosure provides methods of treating cancer by silencing tumor-innervating sensory neurons. The methods include treating cancer by genetic ablation of ion channels (e.g., TRPV1 or NaV1.8), local pharmacological silencing or blockade of neuropeptide release from tumor-innervating nociceptor (e.g., with QX-314 and BoNT/a), as well as the antagonism of the CGRP receptor RAMP1 (e.g., with BIBN 4096).

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: The present invention is directed to wound healing scaffolds cografted with a population of stem cells, wherein the population of stem cells are ABCB5+ stem cells. The scaffolds are, for instance, collagen glycosaminoglycan scaffolds.

Abstract: Provided herein are compositions and methods for determining the structure of individual targets by determining long-range distances within such targets.

Abstract: Methods and compositions comprising ABCB5+ stem cell to treat hyper-inflammatory disorders, such as acute respiratory distress syndrome (ARDS) are provided. Such compositions may be used to treat, for example, patients with severe COVID19.

Abstract: To enable real-time seizure warnings, systems and methods of the present disclosure include a wearable sensor in communication with processors that are configured to receive from the wearable sensor data streams associated with a user that include biomarker data parameters. The processors utilize a seizure forecasting machine learning model to predict a pre-ictal period probability associated with a forecasted time segment based on values of the data streams. The processors determine a segment value for an integration window of a history pre-ictal period probabilities for the forecasted time segment and previously forecasted time segments and determine a pre-ictal period based on the segment value exceeding a pre-ictal probability threshold. The processors determine a pre-ictal risk indication include a seizure treatment administration and cause a computing device to produce the pre-ictal risk indication to indicate a predicted risk of a seizure.

Abstract: Embodiments disclosed here provide engineered modified hematopoietic stem cells (HSCs), artificially prostaglandin E2 (PGE2)-stimulated HSCs, compositions comprising these HSCs, methods of using these modified HSCs for treating autoimmune diseases and disorders and for suppressing the immune system. In particular, the engineered modified HSCs or PGE2-stimulated HSCs express the surface marker, programmed cell death-1 ligand 1 (PD-L1).

Abstract: The present invention is directed to methods and compositions for treating Glioblastoma multiforme (GBM). The risk of developing therapy resistant GBM may be assessed by detecting the presence of ABCB5 in the GBM cells. Therapeutic interventions utilizing an ABCB5 blockade to sensitize the GBM cells to therapeutic agents such as temozolomide are provided.

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: Provided herein, in some aspects, are delivery vehicles comprising a glycosphingolipid and an agent to be delivered attached to the glycosphingolipid. In some embodiments, the glycosphingolipid comprises an oligosaccharide and a short chain (e.g., C0-C3) ceramide. In some embodiments, the agent to be delivered is a therapeutic agent. The glycosphingolipid is able to deliver the agent to a cell or to a cellular compartment, as well as across the musical barrier. In some embodiments, agents delivered using the glycosphingolipid described herein exhibit longer half-life, compared to agents delivered alone. Methods of delivering a therapeutic agent to a subject for treating a disease using the glycosphingolipid delivery vehicle are also provided.

Abstract: Provided herein are methods related to methods of predicting sensitivity to NER-targeting agents for the treatment of cancer. In one aspect, provided herein is a method for treating cancer in a subject) of at least 0.70 and the anti-cancer treatment comprises an alkylating chemotherapeutic agent.

Abstract: Provided herein are Stimulator of Interferon Genes (STING) ligand for use in enhancing immune response and/or as adjuvants in vaccines. In some embodiments, STING ligand is used alone or in combination with Alum in an adjuventation system for early life immunization.

Abstract: The invention provides a method for treating brain or nerve injury.

Abstract: An insertable catheter device includes a shaft including a proximal end and a distal end, an expandable balloon, and an actuator configured to expand and retract the expandable balloon. The actuator includes a fluid conduit that extends through the shaft and is coupled with the expandable balloon to enable inflation and retraction of the expandable balloon via injection or withdrawal of a fluid to or from the expandable balloon via the fluid conduit. The expandable balloon is displaceably retractable into the shaft and extendable from the shaft. A fluid pump is coupled with the fluid conduit to pump the fluid through the fluid conduit. A patch is positioned to be displaced by the expandable balloon when the expandable balloon is inflated, and the expandable balloon is displaceably retractable into the shaft and displaceably extendable from the shaft.

Abstract: The present invention is directed to purified preparations of dermal mesenchymal stem cells that are characterized by the cell surface expression of the ABCB5 P-glycoprotein. The cells may be used for any purpose that mesenchymal stem cells from other course are used. For instance they may be administered to treat an organ transplant recipient to improve allograft survival or as a treatment to patients with autoimmune diseases such as multiple sclerosis and rheumatoid arthritis.

Abstract: The present invention is based on the identification of novel biomarkers predictive of responsiveness to anti-immune checkpoint inhibitor therapies.