Abstract: The invention provides an isolated and purified nucleic acid sequence encoding a chimeric antigen receptor (CAR) directed against B-cell Maturation Antigen (BCMA). The invention also provides host cells, such as T-cells or natural killer (NK) cells, expressing the CAR and methods for destroying multiple myeloma cells.

Abstract: Identification of immunodominant Babesia microti antigens using genome-wide immunoscreening is described. Candidate antigens were screened against sera from patients with clinical babesiosis. Also described are diagnostic assays with high sensitivity and specificity for detecting B. microti-specific antibodies in patient samples using the identified immunodominant antigens.

Abstract: Compounds useful as components of immunogenic compositions for the induction of an immunogenic response in a subject against viral infection, methods for their use in treatment, and processes for their manufacture are provided herein. The compounds comprise a nucleic acid construct comprising a sequence which encodes a Zika virus antigen.

Abstract: Chimeric antigen receptors containing human CD19 antigen binding domains are disclosed. Nucleic acids, recombinant expression vectors, host cells, antigen binding fragments, and pharmaceutical compositions, relating to the chimeric antigen receptors are also disclosed. Methods of treating or preventing cancer in a subject, and methods of making chimeric antigen receptor T cells are also disclosed.

Abstract: The presently disclosed subject matter provides for methods and compositions for enhancing the immune response toward cancers and pathogens. It relates to chimeric antigen receptors (CARs) that specifically target human mesothelin, and immunoresponsive cells comprising such CARs. The presently disclosed mesothelin-targeted CARs have enhanced immune-activating properties, including anti-tumor activity.

Abstract: Disclosed herein are embodiments of a compound useful for treating or preventing betacoronavirus infections such as SARS-Cov-2 infections. Also disclosed is a method for administering the compound to a subject, particularly a human subject, to treat or prevent a betacoronavirus infection in the subject. The compound can comprise an oligomer comprising a nucleic acid base sequence that is antisense to at least a portion of a SARS-CoV-2 genomic RNA, and can comprise a sequence present in the 5? UTR and first 20 nt of coding sequence of the SARS-CoV-2 genomic RNA. The compound also can contain a peptide sequence. In some embodiments, the compound is a peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO).

Abstract: This application provides the first observation of methylmalonylation/malonylation in organic acidemias (OAs), such as methylmalonic acidemia (MMA) and propionic acidemia (PA), which results in modification of enzymes in key pathways dysregulated in OAs, including sirtuin 5 (SIRT5). Hyperacylation of SIRT5 prevents it from de-acylating CPS1 (including removing methymalonyllation), which prevents activation of CPS1 and likewise, inhibits a key component of the glycine cleavage system, GCSH. Based on these observations, provided herein is a mutant form of SIRT5 containing four mutated lysines that cannot accept acyl groups, methods of its use for treating OA patients, and kits.

Abstract: Modified G6PC (glucose-6-phosphatase, catalytic subunit) nucleic acids and glucose-6-phosphatase-? (G6Pase-?) enzymes with increased phosphohydrolase activity are described. Also described are vectors, such as adeno-associated virus (AAV) vectors, and recombinant AAV expressing modified G6Pase-?. The disclosed AAV vectors and rAAV can be used for gene therapy applications in the treatment of glycogen storage disease, particularly glycogen storage disease type Ia (GSD-Ia), and complications thereof.

Abstract: Antibodies and antigen binding fragments that specifically bind to gp120 and neutralize HIV-1 are disclosed. Nucleic acids encoding these antibodies, vectors and host cells are also provided. Methods for detecting HIV-1 using these antibodies are disclosed. In addition, the use of these antibodies, antigen binding fragment, nucleic acids and vectors to prevent and/or treat an HIV-1 infection is disclosed.

Abstract: Provided herein are antisense oligonucleotides (ASOs) specific for Rab13 and Net1, for example specific for a GA-rich region of the 3?-UTR. In some examples, the ASOs are modified. Methods of using these ASOs to reduce the migration of metastatic cancer cells are provided, for example as a use to treat metastatic cancer.

Abstract: The invention provides an isolated and purified nucleic acid sequence encoding a chimeric antigen receptor (CAR) directed against B-cell Maturation Antigen (BCMA). The invention also provides host cells, such as T-cells or natural killer (NK) cells, expressing the CAR and methods for destroying multiple myeloma cells.

Abstract: Disclosed herein are modified Leishmania species and compositions thereof, such as live, attenuated organisms, immunogenic compositions, vaccines, and pharmaceutical compositions. Further disclosed are methods related to the modified Leishmania species, such as methods of production and methods of use.

Abstract: The invention provides an isolated and purified nucleic acid sequence encoding a chimeric antigen receptor (CAR) directed against B-cell Maturation Antigen (BCMA). The invention also provides host cells, such as T-cells or natural killer (NK) cells, expressing the CAR and methods for destroying multiple myeloma cells.

Abstract: An improved antibody-drug conjugate (ADC) targeting CD276-positive tumors is described. The ADC includes a CD276-specific IgG1 antibody having a heavy chain modified to prevent interaction of its Fc domain with endogenous Fc receptors and to introduce a cysteine for site-specific conjugation of the drug. The CD276-specific ADC is capable of potently eradicating CD276-positive tumors in several animal models.

Abstract: Immunogenic compositions are disclosed that include Neisseria meningitidis microvesicles, such as outer membrane vesicles (OMV) and/or blebs, from PorA?PorB? Neisseria, such as PorA?PorB?ampM? Neisseria meningitidis. These immunogenic compositions are of use to induce an immune response to Neisseria, including Neisseria meningitidis and Neisseria gonorrhea.

Abstract: A viral exposure signature (VES) that can identify early stage, pre-symptomatic hepatocellular carcinoma (HCC) among at-risk patients is described. The VES was developed using serological profiling and synthetic virome technology to identify unique viral peptide epitopes corresponding to 61 viral species. Methods of identifying a subject with early stage (pre-symptomatic) HCC using the VES are described.

Abstract: Single-domain monoclonal antibodies that specifically bind B7H3 (also known as CD276) are described. The single-domain antibodies include camel VHH and rabbit VH domain nanobodies selected from phage display libraries. Chimeric antigen receptors (CARs) and other antibody conjugates targeted to B7H3 are also described. The single-domain antibodies and conjugates thereof can be used for the diagnosis and treatment of B7H3 expressing solid tumors.

Abstract: Vaccines that elicit broadly protective anti-influenza antibodies. The vaccines comprise nanoparticles that display HA trimers from Group 2 influenza virus on their surface. The nanoparticles are fusion proteins comprising a monomeric subunit (e.g., ferritin) joined to stabilized stem regions of Group 2 influenza virus HA proteins. The fusion proteins self-assemble to form the HA-displaying nanoparticles. Also provided are fusion proteins, and nucleic acid molecules encoding such proteins, and assays using nanoparticles of the invention to detect anti-influenza antibodies.

Abstract: Methods are disclosed for determining whether a subject has a synucleinopathy. Methods are also disclosed for detecting misfolded alpha synuclein (?Syn) in a biological sample or fraction thereof. These methods include the use of an ?Syn seeding assay.

Abstract: Embodiments of a method for inhibiting viral infection in a subject are provided herein. In some embodiments, the method comprises administration of a competitive antagonist of ouabain binding to ATP1A1 to inhibit respiratory syncytial virus infection in the subject.