Abstract: Described herein are methods and compositions for treating cancer. Aspects of the invention relate to determining if a biological sample obtained from a subject for GSK3a bodies (puncta), e.g., in response to asparaginase treatment, and administering to a subject having cancer an asparaginase and an agent that inhibits GSK3? to a subject who has been identified as forming GSK3?-positive bodies.

Abstract: The present invention is directed to compositions and methods to increase the expression of PD-L1 and/or IDO-1 in a population of cells, the modulated cells expressing increased PD-L1 and/or IDO-1, and methods related to the immunosuppressive effects obtained by cells expressing increased PD-L1 and/or IDO-1.

Abstract: The present invention relates in part to the discovery of genes that are deregulated in cancer stem cells (e.g., melanoma stem cells). In some aspects, methods for treating individuals having melanoma are provided; the methods involve modulating (e.g., inducing, inhibiting, etc.) the activity of the cancer stem cell associated genes. In other aspects, cell surface genes that are upregulated in melanoma stem cells are targeted for the selective isolation, detection, and killing of cancer stem cells in melanoma. Other aspects of the invention relate to reagents, arrays, compositions, and kits that are useful for diagnosing and treating melanoma.

Abstract: Provided herein are methods and compositions 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.

Abstract: Disclosed herein are agents that enhance muscle stem cell engraftment, as well as methods and compositions using the same.

Abstract: The present disclosure provides genetically modified antibody-producing cells comprising edited chromosomal sequences associated with immunoglobulin heavy chain constant region, the IgH locus. In particular, these cells are generated using a CRISPR/Cas 9-mediated editing process. The disclosure also provides specific guide RNA (gRNA) guide sequences that target the chromosomal sequence of immunoglobulin heavy chain constant region in the Switch regions.

Abstract: The present invention features a dual vector system for disrupting and replacing a target gene comprising a mutation (e.g., dominant, recessive mutation). Embodiments of the invention may also provide compositions comprising the dual vector system, and methods of using the dual vector system, including but not limited to methods of modifying the genome of a cell, methods of genomic editing, and methods of treating cells or a subject suffering from a genetic disease comprising a mutation.

Abstract: Embodiments disclosed here are production methods and compositions of engineered immune cells, such as B or T lymphocytes, from limited lineage myeloid progenitor cells, or from pluripotent stem cells, or from multilineage hematopoietic progenitor cells comprising the addition of various cell differentiation transcription factors and inhibiting epigenetic histone methylations in said cells.

Abstract: The invention features compositions and methods for characterizing inflammatory bowel disease and inflammatory bowel disease subtypes, such as well Crohn's disease and ulcerative colitis. In one aspect, the invention provides a panel for characterizing inflammatory bowel disease, the panel including two or more (e.g., 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 20, 25) capture molecules each bound to a substrate, wherein each capture molecule specifically binds a marker polypeptide selected from one or more of the following: CD3, CD4, CDS, CD24, CD25, CD27, CD38, CD44, CD45RA, CD45RO, CD127, CD161, CTLA-4, CXCR3, CCR4, CCR6, CCR7, FOXP3, HLA-DR, IFN?, IL-10, IL-17A, IL-21, IL-22, CD11c, IL23p19, CD66b, CD163, CD44, ckit, CD16, NKp46, AHR, and TNF?, or a polynucleotide encoding said marker polypeptide.

Abstract: In certain embodiments, the disclosure relates to compositions and methods relating to a translation-based gene regulation system that functions in mammalian cells. In certain specific embodiments, the disclosure relates to methods of regulating gene expression via modulating translation termination.

Abstract: The present disclosure features methods and compositions for treating Alzheimer's disease. The disclosed methods comprise administering to a subject having or suspected of having Alzheimer's a hematopoietic stem progenitor cell expressing at least one neuroprotective agent, such as ApoE2, Trem2, and/or a metallothionein.

Abstract: The present application is generally directed to methods for identifying immunogens from organisms and pathogens, and in particular for identifying immunogens which when administered as vaccines elicit a cellular and/or humoral immune response. The present application is also directed to pneumococcal T-cell immunogens, and vaccine compositions comprising one or a combination of pneumococcal immunogens and methods for treating or preventing pneumococcal infections using the vaccines thereof. The present invention also encompasses use of the pneumococcal immunogens for diagnostic purposes to identify a subject with a pneumococcal infection.

Abstract: Provided herein are 2?-O-methyl 3?phosphorothioate (MS)-modified synthetic nucleic acid molecules (single guide RNAs (sgRNAs)) in combination with a base editor as a ribonucleoprotein (RNP) complex for an electroporation-based ex vivo targeted gene disruption of the BCL11A erythroid enhancer's +55, +58, or +62, functional regions. Also provided herein are methods relating to the treatment of hemoglobinopathies by reinduction of fetal hemoglobin levels.

Abstract: Embodiments of the technology described herein are based upon the discoveries that neutrophil extracellular traps (NETs) provide a stimulus for thrombus formation and that NETs are present in stored blood products. Accordingly, some embodiments relate to methods of treating and preventing toxicity of NETs and thrombosis caused by NETs. Additional embodiments are directed towards methods of treating stored blood products to prevent transfusion-related injuries.

Abstract: Described herein are methods of prolonging or reactivating organogenesis in a subject in need thereof (e.g., a subject that has impaired organ function such as a prematurely born infant). The methods comprise increasing the expression or activity of Lin28A or Lin28B proteins, inhibiting the expression or activity of let-7 family microRNAs, and/or inhibiting the expression or activity of Dis3L2 exonuclease.

Abstract: Apparatuses and methods for using Raman Resonance Spectroscopy to evaluate metabolic and oxygenation status of the eye are disclosed herein. In some embodiments, metabolic mapping of the eye may be performed by aligning a Raman spectrum and a recorded spatial image of the eye.

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: Filtration apparatuses, kits, and methods for rapid isolation of intact, viable mitochondria from tissues are described with mitochondria isolated by differential filtration through nylon mesh filters. Mitochondria can be isolated in less than 30 minutes using the filtration apparatuses, kits, and methods described.

Abstract: The present disclosure involves a system and method of use for delivering an interventional instrument, such as an artificial chordae tendineae (ACT) delivery instrument, into a beating heart. By delivering interventional instruments in a beating heart via the system described here, complex and invasive open heart procedures as well as cardiopulmonary bypass can be avoided, significantly reducing patient trauma and recovery time as well as operation time and complexity. The system disclosed includes an optical imaging system to provide the practitioner with visual imagery from the end of the system, and allow for real-time or near real-time imaging of tissue inside the beating heart (e.g., leaflet tissue). This provides for accurate verification of placement of the interventional instrument. In addition, the system includes a grasping mechanism, which allows the practitioner to hold tissue in position prior to placing the interventional instrument.

Abstract: A suturing apparatus and method thereof using autotransfer are provided. The suturing apparatus includes a tube that has a rod movable there within and a needle that has an insertion portion. A suture engages with a tip of the needle or the tube. The insertion portion of the needle is inserted through a material prior to engagement with the suture and is withdrawn with the suture back through the material. The suturing system allows for single hand operation, thus simplifying a suturing process.