Abstract: Use of a rAAV3B vector to deliver gene products to human hepatocytes is described. The rAAV3B vectors achieve high levels of transduction even in the presence of pre-existing immunity to AAV8 or AAVrh10. Compositions and treatment regimens are described. Also provided are rAAV engineered to facilitate purification and methods of purifying the AAV.

Abstract: The present disclosure relates to compositions and methods for predicting cancer survival or toxicity in a subject receiving a chimeric antigen receptor (CAR) T cell therapy. The present disclosure further discloses compositions, e.g., pharmaceutical compositions, and methods for treating said subject.

Abstract: The present invention provides compositions and methods for inhibiting ACSS2 for modulating histone acetylation or for treating or preventing a neurological disease or disorder.

Abstract: In various aspects and embodiments the invention provides compositions and methods useful in the treatment of acute lung injury (ALI).

Abstract: In certain embodiments, this application discloses methods for the diagnosis and prognosis of prostate cancer. In some embodiments, the invention takes advantage of the combinatorial utility of certain biomarkers to prognose and diagnose prostate cancer at an early stage. In certain embodiments, the methods described herein do not require the selection of cells from a particular tissue compartment, and are therefore suitable for analysis of cancer tissue in which compartmentalization is lost.

Abstract: The present disclosure provides monoclonal antibodies that bind ?-Synuclein. In certain aspects, the antibodies preferentially bind to ?-Synuclein fibrils over ?-Synuclein monomer. In other aspects, the invention comprises a method of treating ?-Synucleopathic disease in a subject, comprising administering any of the antibodies of the invention to the subject. In yet other aspects, the invention comprises methods of detecting ?-Synuclein fibrils using any of the antibodies of the invention.

Abstract: A composition comprising at least one AAV vector formulated for central nervous system delivery is described. The composition comprises at least one expression cassette which contains sequences encoding an anti-neoplastic immunoglobulin construct for delivery to the brain operably linked to expression control sequences therefor and a pharmaceutically acceptable carrier. The anti-neoplastic immunoglobulin construct may be an immunoglobulin modified to have decreased or no measurable affinity for neonatal Fc receptor (FcRn). Also provided are methods of using these constructs in preparing pharmaceutical compositions and uses thereof in anti-neoplastic regimens, particularly for primary and/or metastatic cancers of the brain.

Abstract: Compositions and methods for reducing viral load in the oral cavity, particularly Coronavirus such as SARS-CoV-2 and Influenza viral loads are disclosed. Also disclosed are compositions and methods for reducing bacterial or fungal loads in the oral cavity. The compositions comprise a trapping molecule having binding affinity for a protein, glucan, or other molecule on the surface of a virus or microorganism. A carrier appropriate for oral administration, in the form of a chewing gum, long-acting lozenge, or tablet, is further disclosed, to enable easy administration before or after exposure to the infective agent.

Abstract: A bispecific cytotoxic lymphocyte or macrophage-redirecting autoantibody comprising a cytotoxic lymphocyte or macrophage targeting domain and an autoantibody. Methods for producing bispecific cytotoxic lymphocyte or macrophage-redirecting autoantibody comprising a cytotoxic lymphocyte or macrophage targeting domain and an autoantibody. Methods for treating a subject in need thereof comprising administering to the subject an isolated bispecific cytotoxic lymphocyte or macrophage-redirecting autoantibody.

Abstract: A rAAV for treatment of a disorder associated with a deficiency in hypoxanthine-guanine phosphoribosyltransferase (HPRT) enzyme levels via gene delivery is provided herein. Also provided herein is a method for treating disorder associated with a deficiency in hypoxanthine-guanine phosphoribosyltransferase (HPRT) enzyme levels by direct delivery to dopaminergic neurons. Correction of Lesch-Nyhan disease may be monitored by assessing dopamine metabolism levels and/or imaging for the presence of dopaminergic cell bodies.

Abstract: Emerging evidence indicates that diminished activity of the vasoprotective axis of the renin-angiotensin system, constituting angiotensin converting enzyme2 (ACE2) and its enzymatic product, angiotensin-(1-7) [Ang-(1-7)] contribute to pulmonary hypertension (PH). However, clinical success for long-term delivery of ACE2 or Ang-(1-7) would require stability and ease of administration to increase patient compliance. Chloroplast expression of therapeutic proteins enables their bioencapsulation within plant cells to protect from acids and gastric enzymes; fusion to a transmucosal carrier facilitates effective systemic absorption. Oral feeding of rats with bioencapsulated ACE2 or Ang-(1-7) attenuated monocrotaline (MCT)-induced increase in right ventricular systolic pressure, decreased pulmonary vessel wall thickness and improved right heart function in both prevention and reversal protocols.

Abstract: This invention provides purified preparations of an RNA, oligoribonucleotide, or polyribonucleotide comprising a modified nucleoside, and methods of assessing purity of purified preparations of an RNA, oligoribonucleotide, or polyribonucleotide comprising a modified nucleoside.

Abstract: Provided herein is a nucleic acid comprising consensus amino acid sequence of foot-and-mouth disease FMDV VP1-4 coat proteins of FMDV subtypes A, Asia 1, C, O, SAT1, SAT2, and SAT3 as well as plasmids and vaccines expressing the sequences. Also provided herein is methods for generating an immune response against one or more FMDV subtypes using the vaccine as described above as well as methods for deciphering between vaccinated mammals with the vaccine and those that are infected with FMDV.

Abstract: This invention provides methods for treating diseases associated with elevated p38 mitogen-activated protein kinase activity. Moreover, the invention provides methods for testing a candidate compound for a p38 mitogen-activated protein kinase modifying activity by calculating the level of relocalization of an SMN complex component from the cytoplasm to the nucleus of a cell. Additionally, the invention provides a kit and a system for calculating the same.

Abstract: Provided are methods for forming polymer-infiltrated nanoparticle films by using capillary action to draw mobile molecular chains into the pores of a bed of nanoparticles. The chains can spread across the entire bed of nanoparticles. The disclosed methods also provide the formation of patterned polymer-infiltrated nanoparticle film compositions, as well as laterally graded compositions and compositions that feature a polymer gradient through the composition's thickness. Articles can be formed that include a plurality of polymer types infiltrated into the bed of nanoparticles.

Abstract: In one example, a bearing or transmission has an outer race that extends circumferentially about a central axis. An inner race is disposed within the outer race and extends circumferentially about the central axis so as to be coaxial with the outer race. A plurality of load-bearing rolling elements are disposed between the inner race and the outer race, and are spaced circumferentially from one another so as to not contact one another. A plurality of separator rolling elements are disposed within the outer race. Each separator rolling element is disposed between a respective pair of the load-bearing rolling elements such that the separator rolling element is in rolling contact with the load-bearing rolling elements of the respective pair. The load-bearing and separator rolling elements form a contiguous ring.

Abstract: Multi-state switchability is highly desirable in optoelectronic devices. For liquid crystal (LC) based devices, the stability of any configuration is achieved through a balance between imposed interactions and the LC's orientational elasticity. In most cases, the latter acts to resist deformation. By combining surface topography and chemical patterning, provided here are the effects of saddle-splay orientational elasticity, a property that, despite being intrinsic to all LCs, is routinely suppressed. Utilizing theory and continuum elastic calculations, provided here are example conditions for which, even using generic, achiral LC materials, spontaneously broken surface symmetries develop. Also provided are multi-stable devices in which a weak, but directional, applied field switches between spontaneously-polar surface state domains. The disclosed approach is useful in low-field and fast-switching optoelectronic devices, beyond those attainable by current technologies.

Abstract: This disclosure provides a culture media that facilitates survival and/or engraftment of transplanted cells. In one embodiment, this culture media comprises collagen I, albumin, L-glutamine and NaHCO3. In one embodiment, this culture media promotes enhanced survival and engraftment of ?-cells of human pancreatic islets transplanted under the skin. The protective effect of this culture media is mediated, at least partly, by upregulating anti-apoptotic signaling pathways.

Abstract: The present disclosure provides compositions and methods for site-specific labeling of antibodies by proximity-based sortase-mediated ligation. The ligation method utilizes a non-canonical isopeptide ligation reaction catalyzed by newly identified variants of S. aureus sortase A. An antibody binding domain (e.g., protein A or protein G) is fused to the variants of SrtA to bring the enzyme into close proximity of an antibody, thereby significantly increases the efficiency of isopeptide bond formation.

Abstract: The disclosure relates to immune cells comprising one or more vectors comprising a nucleic acid sequence encoding a chimeric antigen receptor specific for CD19 and a nucleic acid sequence encoding an enhancer of T cell priming (e.g., IL-18), compositions comprising the T cells, and methods of generating and/or using the T cells to treat diseases associated with the expression of CD19.