Abstract: The invention provides methods for overcoming glucocorticoid resistance of cancers by inhibition of CASP1. Also disclosed are diagnostic methods for determining glucocorticoid resistance potential by measuring expression level or promoter methylation status of CASP1 gene and/or NLRP3 gene.

Abstract: Provided herein are compositions and methods for accurate and scalable Primary Template-Directed Amplification (PTA) nucleic acid amplification and sequencing methods, and their applications for research, diagnostics, and treatment.

Abstract: The present disclosure relates to chemical compounds that modulate pantothenate kinase (PanK) activity for the treatment of metabolic disorders (such as diabetes mellitus type II), neurologic disorders (such as pantothenate kinase-associated neurodegeneration), pharmaceutical compositions containing such compounds, and their use in treatment. This abstract is intended as a scanning tool for purposes of searching in the particular art and is not intended to be limiting of the present invention.

Abstract: Methods and compositions using an inhibitor of EGFR signaling for prevention or an inhibitor of EGFR signaling and a nucleic acid molecule encoding an atonal-associated factor for treatment of hearing loss are described.

Abstract: The invention provides a chimeric receptor comprising NKG2D, DAP10 and CD3 zeta. Also disclosed is a composition comprising this chimeric receptor and methods for making and using it to enhance the cytotoxicity and antitumor capacity of NK cells. The invention also encompasses methods for use of NKG2D-DAP10-CD3 zeta polypeptides, vectors and cells in methods for treating cancer and other proliferative disorders, as well as infectious diseases.

Abstract: The present disclosure provides chimeric MyD88 receptors. Also provided are polynucleotides encoding the chimeric MyD88 receptors, vectors comprising the polynucleotides encoding the chimeric MyD88 receptors, and cell compositions comprising the chimeric MyD88 receptors, polynucleotides and/or vectors. Pharmaceutical compositions comprising the polypeptides, polynucleotides, vectors, or cells of the present disclosure, and their uses in treating a disease in a subject are also provided.

Abstract: The present invention provides methods of genetically modifying an immune cell such that the immune cell expresses a transgene in an activation dependent manner. The application also provides genetically modified immune cells prepared using such methods, and the uses of the genetically modified immune cells in immunotherapy (e.g., adoptive cell therapy) for treatment of a disease such as cancer, autoimmune disease or infectious disease.

Abstract: The present invention provides a chimeric antigen receptor (CAR) comprising an extracellular target-binding domain comprising at least one glucose-regulated-protein 78 (GRP78)-binding moiety. The present invention further provides polynucleotides and recombinant vectors encoding such CARs. The present invention further provides isolated host cells and methods for preparing isolated host cells expressing the CARs. The present invention further provides pharmaceutical compositions comprising the host cells and methods for treating a tumor using the pharmaceutical compositions.

Abstract: Provided are CDK2-PROTAC compounds and pharmaceutical compositions thereof that can be used for specific degradation of the cyclin-dependent kinase 2 (CDK2) protein, for treatment of cancers and other CDK2 related diseases as well as for prevention and treatment of hearing loss.

Abstract: Methods for detecting the presence of proteins in a subject are described. The proteins detected can be indicative of acute myeloid leukemia (AML). The proteins can be particularly useful for monitoring minimal residual disease (MRD) in AML.

Abstract: Methods and formulations are provided for treating spinobulbar muscular atrophy in a subject in need thereof. By administering a therapeutically effective amount of a selective androgen receptor modulator or a small molecule such as 1-[2-(4-methylphenoxy)ethyl]-2-[(2-phenoxyethyl)sulfanyl]-1H-benzimidazole or a derivative, prodrug, or pharmaceutically acceptable salt thereof, one or more symptoms of spinobulbar muscular atrophy can be ameliorated. The effective amount can be effective to prevent or delay loss of body weight, a loss of mobility, and/or a loss of physical strength in the subject; to prevent or delay neurogenic atrophy and/or to prevent a loss of spinal cord motor neurons in the subject; to restore the frequency of type I myofibers to normal levels for a healthy subject; and/or to reverse testicular atrophy in the subject.

Abstract: The present invention relates to a chimeric receptor capable of signaling both a primary and a co-stimulatory pathway, thus allowing activation of the co-stimulatory pathway without binding to the natural ligand. The cytoplasmic domain of the receptor contains a portion of the 4-1BB signaling domain. Embodiments of the invention relate to polynucleotides that encode the receptor, vectors and host cells encoding a chimeric receptor, particularly including T cells and natural killer (NK) cells and methods of use.

Abstract: The present invention provides a chimeric antigen receptor (CAR) comprising an extracellular target-binding domain comprising a B7-H3 binding moiety. The present invention further provides polynucleotides and recombinant vectors encoding such CARs. The present invention further provides isolated host cells and methods for preparing isolated host cells expressing the CARs. The present invention further provides pharmaceutical compositions comprising the isolated host cells and methods for treating a tumor using the pharmaceutical compositions.

Abstract: Provided herein are compositions and methods for accurate and scalable Primary Template-Directed Amplification (PTA) nucleic acid amplification and sequencing methods, and their applications for research, diagnostics, and treatment.

Abstract: Aspects of the disclosure relate to compositions and methods for predicting prognosis and classifying risk of subjects having certain cancers, for example acute myeloid leukemia (AML). In some embodiments, methods described by the disclosure comprise a step of assessing the mRNA expression of certain leukemic stem cell (LSC)-enriched genes in a subject to produce a predictive score for pediatric AML. In some embodiments, methods described by the disclosure comprise a step of assessing the mRNA expression of certain genes of pharmacological relevance for standard chemotherapy consisting of Cytarabine (also known as Ara-C), daunorubicin and etoposide in a subject to produce a predictive score for pediatric AML.

Abstract: The present invention provides chimeric cytokine receptors, particularly chimeric cytokine receptors that canbe activated in tumor microenvironment, and their uses in tumor immunotherapy (e.g., adoptive cell therapy). The present invention further provides methods of genetically modifying therapeutic cells resulting in an enhanced immune response against a target antigen. The application further provides therapeutic cells that express said chimeric cytokine receptors and methods for treating patients using the modified therapeutic cells.

Abstract: The present invention provides, in certain aspects, a natural killer (NK) cell that expresses all or a functional portion of interleukin-15 (IL-15), and methods for producing such cells. The invention further provides methods of using a natural killer (NK) cell that expresses all or a functional portion of interleukin-15 (IL-15) to treat cancer in a subject or to enhance expansion and/or survival of NK cells.

Abstract: The present disclosure relates to methods of treating a coenzyme A reduction, elevation, sequestration, toxicity, or redistribution (CASTOR) disease such as, for example, defects in fatty acid oxidation enzymes, methylmalonic acidemia, glutaric acidemia, propionic academia, and HMG-CoA lyase, via small molecule modulators of CoA levels. This abstract is intended as a scanning tool for purposes of searching in the particular art and is not intended to be limiting of the present invention.

Abstract: The application provides T cell gene expression signatures that can be used to predict T cell therapy outcomes.

Abstract: Described is a method for treating a social memory deficit in a subject with a neuropsychiatric disease is treated by administering a peptide encoded by 2510002D24Rik or Atp23 genes, a vector expressing such peptide, or an agent capable of increasing the level or activity of such peptide. The method may be used to treat schizophrenia or autism. The peptide, vector or agent can be administered directly to the hippocampus, such as by transcranial surgical injection.