Abstract: The invention provides a chimeric antigen receptor (CAR) comprising an antigen binding domain specific for TSLPR, a transmembrane domain, and an intracellular T cell signaling domain. Nucleic acids, recombinant expression vectors, host cells, populations of cells, antibodies, or antigen binding portions thereof, and pharmaceutical compositions relating to the CARs are disclosed. Methods of detecting the presence of a proliferative disorder, e.g., cancer, in a mammal and methods of treating or preventing a proliferative disorder, e.g., cancer, in a mammal are also disclosed.

Abstract: Heptamethine cyanines for use as fluorescent markers of the biliary system are disclosed. Certain heptamethine cyanines exhibit biliary system specificity and methods for in vivo visualization of a biliary system of a subject are provided. The methods may be for diagnostic purposes and/or for visualization of biliary systems during surgery.

Abstract: The present disclosure provides adeno-associated viral vectors, recombinant adeno-associated virus (rAAV) and methods of using such vectors and viruses in gene therapy for treating methylmalonic acidemia in patients with methylmalonyl-coA mutase (MVMUT) deficiency. Also provided are pharmaceutical compositions comprising recombinant adeno-associated virus (rAAV) and a pharmaceutically acceptable carrier or excipient.

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 devices for recovering cryopreserved tissue can comprise a receptacle that receives a sealed, frozen tissue container containing cryopreserved tissue and cryopreservation media, with at least one recovery media chamber and a waste material chamber fluidly coupled to the tissue container receptacle. The recovery device can be inserted into a regulator apparatus that facilitates thawing and warming of the media and tissue, and regulation of the flow of recovery media through the tissue container to flush out the thawed cryopreservation media into the waste chamber. The regulator can identify the tissue based on an ID tag on the tissue container and automatically apply an appropriate algorithm for thawing, culturing, and maintaining the tissue in a viable state.

Abstract: The invention is directed to methods of treating or preventing a Rhabdoviridae virus infection in a mammal comprising administering griffithsin, or a fragment or mutant thereof, to the mammal.

Abstract: Disclosed is an isolated or purified T cell receptor (TCR), wherein the TCR has antigenic specificity for a mutated human RAS amino acid sequence with a substitution of glycine at position 13 with aspartic acid. The TCRs may recognize G13D RAS presented by an HLA-DQ heterodimer. Related polypeptides and proteins, as well as related nucleic acids, recombinant expression vectors, host cells, populations of cells, and pharmaceutical compositions are also provided. Also disclosed are methods of detecting the presence of cancer in a mammal and methods of treating or preventing cancer in a mammal.

Abstract: Provided is a method of treating cancer, particularly cancers associated with an overexpression of polo-like kinase (Plk1), comprising administering a compound of formula (I) or a pharmaceutically acceptable salt thereof in which ring A, X1, X2, X3, X4, X5, R2, R3, R4, n, bond a, and bond b are described herein. Exemplary compounds of formula (I) and pharmaceutically acceptable salts thereof, especially those that selectively inhibit the polo box domain of Plk1, also are provided.

Abstract: Methods for the rapid amplification of extremely low quantity nucleic acids in a sample are provided. The disclosed methods are capable of amplifying less than 1 pg of DNA and/or RNA from a biological sample using a single tube and one-step or two-step preparation.

Abstract: The present disclosure is directed to antibodies binding to Glypican 2 and methods of using such antibodies to treat cancers that express or overexpress the Glypican 2 antigen.

Abstract: The invention is directed to a more efficient lentiviral vector comprising a nucleic acid sequence encoding a human ?-globin protein or a human ?-globin protein, which is oriented from 5? to 3? relative to the lentiviral genome. The invention also provides a composition and method utilizing the lentiviral vector.

Abstract: The present disclosure is directed to methods and compositions for the diagnosis and/or treatment of tumors, such as ocular tumors, using virus-like particles conjugated to photosensitive molecules.

Abstract: Methods are disclosed herein for treating glaucoma in a subject. In some embodiments, the methods increase retinal ganglion cell survival. The disclosed methods use exosomes and/or miRNA.

Abstract: Disclosed are inhibitors of the plasmodial surface anion channel (PSAC) inhibitors and the use thereof in treating or preventing malaria in an animal such as a human, comprising administering an effective amount of an inhibitor or a combination of inhibitors. An example of such an inhibitor is a compound of formula I, or a pharmaceutically acceptable salt thereof, wherein R1 to R7 are as described herein.

Abstract: Monoclonal antibodies that specifically bind gp42 of Epstein-Barr virus (EBV) are described. The antibodies are capable of blocking fusion of EBV-infected cells and neutralizing EBV infection. Use of the EBV-specific monoclonal antibodies, and conjugates thereof, for the treatment and prophylaxis of EBV infection is also described.

Abstract: Featured are methods, apparatus and devices for detecting a hematoma in tissue of a patient. In one aspect, such a method includes emitting near infrared light continuously into the tissue from a non-stationary near infrared light emitter and continuously monitoring the tissue using a non-stationary probe so as to continuously detect reflected light. The near infrared light is emitted at two distances from a brain of the patient, so the emitted light penetrates to two different depths. Such a method also includes applying a ratiometric analysis to the reflected light to distinguish a border between normal tissue and tissue exhibiting blood accumulation.

Abstract: Nucleic acid constructs encoding a chimeric antigen receptor (CAR) and a truncated human epidermal growth factor receptor (huEGFRt) are described. The encoded CARs include a tumor antigen-specific monoclonal antibody, such as a glypican-3 (GPC3)-specific, a GPC2-specific or a mesothelin-specific monoclonal antibody, fused to a CD8? hinge region, a CD8? transmembrane region, a 4-1BB co-stimulatory domain and a CD3? signaling domain. Isolated host cells, such as isolated T cells that co-express the disclosed CARs and huEGFRt are also described. T cells transduced with the disclosed CAR constructs can be used for cancer immunotherapy.

Abstract: Disclosed are powered gait assistance systems that include a controller, sensors, and a motor coupled to a patient's thigh and lower leg and operable to apply assistive torque to the patient's knee joint to assist the patient's volitional knee pivoting muscle output during selected stages of the patient's gait cycle, such that the assistive torque applied by the motor improves the patient's leg posture, muscle output, range of motion, and/or other parameters over the gait cycle. The sensors can include a torque sensor that measures torque applied by the motor, a knee angle sensor, a foot sensor that measures ground contact of the patient's foot, and/or other sensors. The controller can determine what stage of the patient's gait cycle the patient's leg is in based on sensor signals and cause the motor to apply corresponding assistive torque to the knee based on the gait cycle stage, sensor inputs, and known patient characteristics.

Abstract: Optimized chimeric antigen receptors (CARs) targeting glypican-1 (GPC1) that include a 12-amino acid hinge region from IgG4 are described. The optimized CARs include a transmembrane domain from either CD8 or CD28. Immune cells, such as T cells or natural killer cells, expressing the optimized CARs can be used to treat GPC1-positive solid tumors.

Abstract: Embodiments of immunogens based on the HIV-1 Env fusion peptide and methods of their use and production are disclosed. Nucleic acid molecules encoding the immunogens are also provided. In several embodiments, the immunogens can be used to generate an immune response to HIV-1 Env in a subject, for example, to treat or prevent an HIV-1 infection in the subject.