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 disclosure provides compositions and methods for delivery of therapeutic agents across a barrier. The compositions include a therapeutic agent (e.g., antimicrobial agent, antibiotic), a permeation enhancer comprising sodium dodecyl sulfate, limonene, and/or bupivacaine which increases the flux of the therapeutic agent across the barrier, and a matrix forming agent that is a poloxamer (e.g., poloxamer P188), wherein the composition comprises: between about 0.2% and 3.2% wt/vol of a permeation enhancer that is sodium dodecyl sulfate; when present, between about 0.2% and 2.0% wt/vol of a permeation enhancer that is bupivacaine; between about 0.5% and 7.0% wt/vol of a permeation enhancer that is limonene; between approximately 18-62% P188; and the composition forms a gel at temperatures above a phase transition temperature that is less than about 37° C. The matrix forming agent forms a gel at a suitable gelation temperature and rheological properties for use in drug delivery.

Abstract: Embodiments herein provide specially designed synthetic BCL11A-targeting microRNAs for RNA polymerase II expression, and methods of use to treat hemoglobinopathies such as sickle cell disease or thalassemia by increasing the expression levels of fetal hemoglobin levels.

Abstract: Provided herein, in some aspects, are compositions comprising vasoocclusion-inhibiting agents encapsulated in RBCs and uses thereof for treating blood vessel occlusion (e.g., in Sickle Cell Disease).

Abstract: Provided herein are imidazopyrimidine compounds, such as compounds of Formula (I), for use in enhancing human immune response and/or as adjuvants in vaccines.

Abstract: Provided herein are materials and methods for efficiently delivering nucleic acids to cochlear and vestibular cells, and methods of treating sensory transduction disorders associated with a genetic defect. Some embodiments are directed to a synthetic inner ear hair cell targeting adeno-associated virus (AAV) vector, a cell comprising the synthetic inner ear hair cell targeting AAV vector, and method of treating Usher Syndrome in a subject using the synthetic inner ear hair cell targeting AAV vector.

Abstract: The present disclosure relates to compositions and methods for treating Williams syndrome (WS), herein identified as a neurodevelopmental oligodendrocyte hypomyelination-associated disease, and to compositions and methods for treatment of other neurodevelopmental myelination abnormality diseases or disorders.

Abstract: The methods and uses described herein relate to the modulation of the immune system by modulation of Sema3F levels and/or activity, e.g. suppressing allograft rejection or inflammation by administering a Sema3F agonist or increasing an immune response by administering a Sema3F inhibitor.

Abstract: The present invention features, in some embodiments, compositions comprising ex vivo or in vitro generated functional enteroendocrine (EE) cells, or enteroids, rectoids, or organoids comprising functional enteroendocrine cells, and methods of obtaining and using such cells for treating metabolic and gastrointestinal diseases, disorders, or conditions.

Abstract: Provided herein are methods and compositions related to treating a hemoglobinopathy. Also provided herein a gene therapeutics for the treatment of hemoglobinopathies and the induction of fetal hemoglobin (HbF).

Abstract: A method of detecting triple negative breast cancer (TNBC) is provided. Overexpression of ICAM-1 is linked to an increased risk of TNBC. A composition of matter is also provided that binds an anti-ICAM~1 antibody to a nanoparticle. The composition may be used as an imaging agent and/or a therapeutic targeting agent. A therapeutically active molecule may be bound to the composition to provide targeted therapy.

Abstract: The inventors have developed tools for quantifying the mitochondrial redox state of in vivo, in situ tissue using resonance Raman spectroscopy. The tissue is illuminated with an excitation beam that causes the tissue to scatter Raman-shifted light, which is collected and analyzed to produce coefficients representing the relative concentrations of different chromophores in the tissue. These relative concentrations indicate the redox state of whole mitochondria, hemoglobin oxygen saturation, myoglobin oxygen saturation, and/or redox state of individual cytochrome complexes in mitochondria of the in vivo, in situ tissue. Quantifiable information about these states and/or saturations can be used to assess tissue health, including organ (dys)function before, during, and after surgery. For example, this information can be used to predict impending cardiac failure, to guide surgical interventions, to monitor organ health after transplantation, or to guide post-operative care.

Abstract: Provided herein are products and methods for detecting analytes using polymers that bind to such analytes and thereby undergo a conformational change or contribute to a newly formed complex.

Abstract: A stent assembly for insertion into an airway of a patient includes a stent defining an interior pathway along a length of the stent. The stent assembly includes a removal instrument for removal of the stent from the airway of the patient. The removal instrument is configured to cause a helical motion of the stent to remove the stent from the airway of the patient.

Abstract: The present invention features methods and composition directed to treating neuronal injury, CNS lesion, and/or promoting axon regeneration using a phosphine sulfide-stabilized phosphine.

Abstract: Provided herein are nucleic acid molecules that target the BCL11A enhancer functional regions, compositions comprising the nucleic acid molecules and methods for increasing fetal hemoglobin levels in a cell by disrupting BCL11A expression at the genomic level. Also provided herein are methods and compositions relating to the treatment of hemoglobinopathies by reinduction of fetal hemoglobin levels. In particular, the nucleic acid molecules target the +62, +58, and/or the +55 enhancer functional regions.

Abstract: Provided herein are compositions comprising VHH conjugates and their uses in treating diseases.

Abstract: Embodiments herein relate to in vitro production methods of hematopoietic stem cell (HSC) and hematopoietic stem and progenitor cell (HSPC) that have long-term multilineage hematopoiesis potentials upon in vivo engraftment. The HSC and HSPCs are derived from pluripotent stem cells-derived hemogenic endothelia cells (HE) by non-integrative episomal vectors-based gene transfer.

Abstract: Described herein are methods and compositions for treating a coronavirus infectious disease, e.g., COVID-19. Aspects of the invention relate to administering to a subject an agent that targets Notch4.

Abstract: The present invention relates to the role of purinergic receptors and ATP in T cell activation and autocrine system signaling. In one embodiment, the present invention provides a method of preventing or treating diabetes by administering a therapeutically effective inhibitor of ATP to a subject. In another embodiment, the present invention provides a method of preventing or treating fibrosis by administering a P2X7R soluble fusion protein. In another embodiment, the present invention provides a method of preventing or treating graft rejection by administering an inhibitor of P2X receptor signaling.