Abstract: The present invention relates to gene therapy delivery and expression systems comprising at least one helper-dependent adenoviral vector containing a nucleic acid sequence encoding for proteoglycan 4 (PRG4) or a biologically active fragment thereof. The invention further relates to a pharmaceutical composition comprising a therapeutically effective amount of at least one helper-dependent adenoviral vector containing said nucleic acid sequence encoding for proteoglycan 4 (PRG4), or a homolog thereof from any other species, or a biologically active fragment thereof. The invention also relates to the use of the novel gene therapy delivery and expression system according to the invention for use in the prevention and/or treatment of camptodactyly-arthropathy-coxa vara-pericarditis (CACP), or a musculoskeletal disorder such as a joint disorder or joint disease.

Abstract: Embodiments of the disclosure encompass adoptive immunotherapy related to cells expressing multiple chimeric antigen receptors (CARs). In specific embodiments, T cells express a HER2-specific CAR, an IL13 R?2-specific CAR, and an EphA2-specific CAR. In particular embodiments, the cells are utilized for cancer treatment, including for glioblastoma.

Abstract: Embodiments of the disclosure encompass immunotherapy for Hepatitis B viral (HBV) infection in an individual in need thereof. The immunotherapy comprises one or more chimeric antigen receptors (CAR) that target a HBV antigen, including CAR molecules that utilize specific scFv antibodies. In certain cases, the CAR comprises one or more mutations to reduce binding to Fc receptors. In specific aspects, cells that express the CAR(s) have reduced cytotoxicity that is safer and/or beneficial to individuals that are immunocompromised.

Abstract: Chromatin 3D structure modulating agents in the context of the present invention are intended to interfere or manipulate the function of loop anchor motifs, such as CTCF motifs. In certain example embodiments, the present invention may block formation of an loop anchor or chromatin domain or induce formation of a loop anchor or chromatin domain at a targeted genomic location. For instance, a loop anchor motif can be altered, such as by mutating (including inverting) a binding motif so as to remove such a motif, or by adding new binding motifs in new locations within a loop domain, so as to reduce the size of an existing loop, so as to modify the size of an existing loop, or combinations thereof. Alternatively, the chromatin 3D structure modulating agent may bind a target region and mask a loop anchor motif, thereby preventing a loop anchor or chromatin domain from forming. The chromatin 3D structure modulating agent may bind a target region and cause a loop anchor of chromatin domain to form.

Abstract: One aspect of the invention provides a flow-diverting system including: a first stent having a proximal end, a distal end and a first sidewall opening; and a second stent having a proximal end, a distal end and a second sidewall opening. The first sidewall opening is of sufficient size for the distal end of the second stent to pass from inside the first stent through the first sidewall opening. The second sidewall opening is of sufficient size for fluid flow from inside the second stent through the second sidewall opening into the first stent.

Abstract: Embodiments of the disclosure concern methods of identifying whether or not antigens from a particular pathogen are immunogenic, including the order of their immunogenicity. Other embodiments concern correlations between attributes of T cells and their clinical efficacy, such as mathematical representations thereof.

Abstract: Wireless treatment of arrhythmias. At least some of the example embodiments are methods including: charging a capacitor of a first microchip device abutting heart tissue, the charging by harvesting ambient energy; charging a capacitor of a second microchip device abutting the heart tissue, the charging of the capacitor of the second microchip device by harvesting ambient energy; sending a command wirelessly from a communication device outside the rib cage to the microchip devices; applying electrical energy to the heart tissue by the first microchip device responsive to the command, the electrical energy applied from the capacitor of the first microchip device; and applying electrical energy to the heart tissue by the second microchip device responsive to the command to the second microchip device, the electrical energy applied from the capacitor of the second microchip device.

Abstract: Embodiments of the invention include methods of treating, preventing, and/or reduce the risk or severity of a condition selected from the group consisting of muscle wasting, muscle weakness, cachexia, and a combination thereof in an individual in need thereof. In some embodiments, particular small molecules are employed for treatment, prevention, and/or reduction in the risk of muscle wasting. In at least particular cases, the small molecules are inhibitors of STAT3.

Abstract: Embodiments of the disclosure encompass highly sensitive and quantitative methods for single-cell sequencing of total RNA. In particular cases, methods utilize annealing of multiple primers to RNA, polytailing of single stranded DNA reverse transcribed therefrom, and utilization of bar codes in primers for amplification of amplicons produced from second strand synthesis.

Abstract: The present disclosure relates to a novel process for expanding T cells, such as autologous T cells, cell populations therefrom, pharmaceutical compositions comprising the said cell populations and use of the cells and compositions for treatment, particular the treatment or prophylaxis of virus infection and/or cancer, for example in immune compromised or immune competent human patients.

Abstract: Embodiments of the disclosure concern methods and compositions related to manipulation of the microbiome in an individual having at least one social behavior deficit. In particular embodiments, an individual has at least one social behavior deficit and was born from a mother who during pregnancy was obese, overweight, or on a high-fat diet during pregnancy or carries mutations associated with neurodevelopmental disorders. In specific embodiments, the individual having at least one social behavior deficit is provided an effective amount of Lactobacillus reuteri for the improvement of at least one symptom of a social behavior deficit.

Abstract: Disclosed herein are compounds and methods for decreasing MECP2 mRNA and protein expression. Such compounds and methods are useful to treat, prevent, or ameliorate MECP2 associated disorders and syndromes. Such MECP2 associated disorders include MECP2 duplication syndrome.

Abstract: One aspect of the invention provides a flow-diverting system including: a first stent having a proximal end, a distal end and a first sidewall opening; and a second stent having a proximal end, a distal end and a second sidewall opening. The first sidewall opening is of sufficient size for the distal end of the second stent to pass from inside the first stent through the first sidewall opening. The second sidewall opening is of sufficient size for fluid flow from inside the second stent through the second sidewall opening into the first stent.

Abstract: The present invention concerns methods of generating CTLs that are able to target at least one antigen from two or more viruses. The method includes exposing mixtures of peptides for different antigens to the same plurality of PBMCs and, at least in certain aspects, expanding the cells in the presence of IL4 and IL7.

Abstract: Embodiments of the technology developed are a non-contact air esthesiometer used for measuring corneal sensitivity. In certain embodiments, the apparatus takes the OKI DX-255 Basic Digital Fluid Dispenser and modifies it for use to produce a 2-second stream of room-temperature air directed at the center of a patient's cornea. The input to the device is a compressed air tank, IN which can be easily changed, connected to an inline filter. The output is a hose line connected to a valve that permits finer adjustments in airflow rate to a disposable 200-microliter-filter pipette tip. This outlet tip is secured with self-setting rubber and housed in a metal stand with horizontal and vertical travel that can be directly mounted to a standard slit lamp. Four red LED lights were placed around the air outflow that can be used for patient fixation and alignment on the central cornea.

Abstract: The application relates to a chimeric antigen receptor that targets Eph receptors and allows activation of co-stimulatory pathways. The application also relates to polynucleotides that encode the chimeric antigen receptor, vectors, and host cells comprising the chimeric antigen receptor. The application also relates to methods for preparing host cells comprising a chimeric antigen receptor in order to improve the in vivo effector function of the chimeric antigen receptor host cells.

Abstract: Embodiments of the disclosure concern methods and compositions related to cancer therapy using myeloid derived suppressor cells (MDSC) as a solo therapy or an adjunct therapy. The MDSCs are prepared by exposing bone marrow cells or blood cells to one or more compositions that induce their differentiation to MDSCs and also to TGF-?1, and in specific embodiments the exposure to TGF-?1 results in the MDSCs having anti-tumor activity and/or immune stimulatory activity.

Abstract: The present invention relates to compositions and methods of treating lysosomal storage diseases and methods of using trehalose.

Abstract: Polypeptides comprising: (i) an MHC class I ? polypeptide association domain, (ii) a transmembrane domain, and (iii) a signalling domain comprising an ITAM-containing sequence are disclosed. Also disclosed are nucleic acids and expression vectors encoding, compositions comprising, and methods using such polypeptides.

Abstract: Provided herein are methods for cell therapy by modifying transfused cells to express an inducible caspase 9 protein, so that the cells may be selectively killed if the patient experiences dangerous side effects. Provided also within relates in part to methods for preventing or treating Graft versus Host Disease by modifying T cells before administration to a patient, so that they may be selectively killed if GvHD develops in the patient.